Disease therapeutics. Medical search. Frequent questions

The first investment by the Accelerator was made in September 2023 in conjunction with the creation of AlveoGene, a new U.K. company launched to develop innovative inhaled gene therapies and transform outcomes in patients with rare respiratory diseases. (ox.ac.uk)
REHOVOT and RAMAT GAN , Israel , Nov. 21, 2023 /PRNewswire/ -- Skip Therapeutics (" Skip "), developer of computational platform for design of Antisense Oligonucleotide (ASO) based therapies and Sheba Medical Center (" Sheba "), one of the leading medical centers in the world, announced today that they have entered a collaboration for the development of ASO-based treatments for rare genetic diseases. (ktla.com)
29, 2023-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, and Voyager Therapeutics, Inc. (Nasdaq: VYGR), today announced the parties have entered into a definitive license agreement for a potential treatment of prion disease. (sangamo.com)
NEW HAVEN, Conn., March 09, 2023 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced its financial results for the fourth quarter and full year ended Dec. 31, 2022 and provided an update on key strategic initiatives. (globenewswire.com)

Skip Therapeutics employs its proprietary bioinformatic engine to identify the most promising ASO-based molecular strategy for treatment of each patient sub-population based on their specific mutations. (ktla.com)
Dr. Dan Dominissini , deputy director of the Center for Cancer Research and director of the Hematology Laboratory at Sheba Medical Center, stated, "We are delighted to collaborate with Skip Therapeutics for the development of cutting edge ASO-based therapies for genetic diseases. (ktla.com)
Skip Therapeutics' novel computational engine will enables us not only to determine the most appropriate therapeutic design for a given genetic disease, but also to directly identify treatable patient populations, thereby maximizing efficiency and minimizing costs. (ktla.com)

Takeda is a leading global biopharmaceutical company with a strong focus in neuroscience and a history of developing transformative therapies across a range of challenging diseases," said Bill Haney , co-founder and chief executive officer of Skyhawk. (biospace.com)
CAMBRIDGE, Mass., June 10, 2022 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc ® FULC , a clinical stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced clinical proof-of-concept data from the ongoing Phase 1b trial of FTX-6058 for the treatment of sickle cell disease (SCD). (benzinga.com)
Avalo Therapeutics , formerly Cerecor is a biopharmaceutical company that develops and commercializes novel products in the fields of neurology and pediatrics. (pharmaceutical-technology.com)
TScan Therapeutics, Inc. (NASDAQ: TCRX) is engaged as a clinical-stage biopharmaceutical company, which is focused on the research and development of T cell receptor (TCR)-based T cell therapies for the treatment of cancer. (financialcontent.com)
Amgen, a leading biopharmaceutical company, will take on the responsibility of developing therapeutics based on the targets identified by TScan. (financialcontent.com)
Wilson Therapeutics is a biopharmaceutical company, based in Stockholm, Sweden, that develops novel therapies for rare diseases. (wikipedia.org)

Newswise - CAMBRIDGE, Mass., Nov. 11, 2020 - Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced that it has received a grant from the Bill & Melinda Gates Foundation to research in vivo sickle cell disease (SCD) treatments using its CRISPR/Cas9 genome editing technology. (newswise.com)
April 23, 2020 /PRNewswire/ -- SwanBio Therapeutics, a gene therapy company developing leading-edge medicines to deliver dramatic clinical efficacy for the treatment of AMN and other neurological diseases, today announces the successful completion of a $52 million expanded Series A financing. (prnewswire.com)
Saturday, February 29 th was Rare Disease Day 2020, and HDSA joined with the community across borders and diseases to raise awareness about rare disorders like HD through our # LetsTalkAboutHD campaign. (hdsa.org)
BlueRock Therapeutics shared a news release on January 7, 2020 announcing the funding of a two-year trial using adult donor stem cells. (parkinson.ca)
NEW YORK , Dec. 17, 2020 /PRNewswire/ -- The National Kidney Foundation (NKF), representing the more than 37 million adults in the U.S. with kidney disease, their families, and the professionals who care for them, believes that a key principle of ethical vaccine allocation must be that the vaccine is made available to patients at the highest risk of severe outcomes from COVID-19 infection . (prnewswire.com)

Thank you for sharing this Journal of Pharmacology and Experimental Therapeutics article. (aspetjournals.org)
Message Body (Your Name) thought you would be interested in this article in Journal of Pharmacology and Experimental Therapeutics. (aspetjournals.org)

Blackstone ( NYSE: BX ) and Alnylam Pharmaceuticals, Inc. ( Nasdaq: ALNY ), the leading RNAi therapeutics company, today announced the closing of the R&D funding component of the companies previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics. (webwire.com)
Alnylam ( Nasdaq: ALNY ) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious, and central nervous system (CNS)/ocular diseases. (webwire.com)
Shares of TScan Therapeutics are skyrocketing after Amgen (NASDAQ: AMGN) and TScan Therapeutics have announced a multi-year collaboration to identify antigens recognized by T cells in patients with Crohn's disease. (financialcontent.com)
The post TScan Therapeutics (NASDAQ: TCRX) and Amgen (NASDAQ: AMGN) Join Forces to Battle Crohn's Disease appeared first on Spotlight Growth . (financialcontent.com)
Neumora Therapeutics plans to list on the Nasdaq under the symbol NMRA . (renaissancecapital.com)
On April 29, 2016, Wilson Therapeutics announced its initial public offering on Nasdaq Stockholm and had its first day of trading on May 12, 2016. (wikipedia.org)

NEW YORK - June 30, 2021 (GLOBE NEWSWIRE) - LEXEO Therapeutics, a clinical-stage gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of Friedreich's ataxia (FA). (curefa.org)

Pharmacology and Therapeutics. (westminster.ac.uk)

The event was hosted by the Oxford-Harrington Rare Disease Centre (OHC), a partnership established in 2019 between the University of Oxford and Harrington Discovery Institute. (ox.ac.uk)
WALTHAM, Mass. , May 6, 2019 /PRNewswire/ -- Skyhawk Therapeutics , Inc. (Skyhawk) today announced a strategic collaboration with Takeda Pharmaceutical Company Limited (Takeda) in which Skyhawk will use its SkySTAR ™ technology platform to discover and pre-clinically develop innovative small molecule treatments directed to certain neurological disease targets. (biospace.com)
2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations. (nih.gov)

Hereditary emphysema (HE) is a form of chronic obstructive pulmonary disease (COPD) that develops as a consequence of a deficiency of AAT in the circulation. (ddw-online.com)
In the higher age groups, the debilitating effects of chronic obstructive pulmonary disease (COPD) and chronic bronchitis represent significantly higher causes of morbidity and mortality. (ddw-online.com)
Asthma and chronic obstructive pulmonary disease. (medlineplus.gov)

Takeda aims to discover and deliver life-transforming treatments in core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. (bioindustry.org)
Recent years have seen unprecedented investment in research and development for countermeasures for high-threat pathogens, including specific and ambitious objectives for development of diagnostics, therapeutics, and vaccines. (cdc.gov)
As antibody-based approaches are identified and advanced, promising small-molecule antivirals currently in clinical stage development should continue to be evaluated for filovirus diseases , with consideration of their added value in combination approaches with bundled supportive care, their penetration in tissues of interest, the absence of interaction with glycoprotein -based vaccines , and filoviral breadth. (bvsalud.org)
In addition, the rapid development of COVID-19 vaccines gives hope that similar technologies will form the basis for devising novel prevention strategies for other infectious diseases that lead to sepsis in both high- and low-resource settings around the world. (cdc.gov)

Mysthera Therapeutics AG has launched to develop first-in-class, oral therapeutics to treat complex autoimmune diseases. (bioworld.com)
This review aims to provide an assessment of the nature and extent of complement involvement in TMA associated with autoimmune diseases such as systemic lupus erythematosus, antiphospholipid syndrome, and scleroderma renal crisis. (jrheum.org)
10 This review aims to provide an assessment of the nature and extent of complement involvement in the underlying pathophysiology of TMA associated with autoimmune diseases that will help to stratify patients for targeted therapy. (jrheum.org)
Sanofi has its UK R&D base on the Babraham Research Campus, south of Cambridge, which is focused on the discovery of novel therapeutics for the treatment of chronic inflammatory and autoimmune diseases, and also brings expertise around antibody discovery and antibody engineering. (bioindustry.org)

Skyhawk's novel approach in correcting RNA expression will complement our expertise in small molecule science and may improve our chances of targeting neurological diseases that have previously been considered undruggable. (biospace.com)
Skyhawk Therapeutics is committed to discovering, developing and commercializing therapies that use its novel SkySTAR ™ ( Skyhawk Small molecule Therapeutics for Alternative splicing of RNA) platform to build small molecule drugs that bring breakthrough treatments to patients. (biospace.com)
In contrast to current anti-inflammatory respiratory therapeutics, certain small molecule and protein protease inhibitors also have the capacity to inhibit directly the chronic airway remodelling and lung degeneration mediated by uncontrolled proteolytic activity. (ddw-online.com)
Shunichi Kashida Ph.D., Representative Director and CEO at xFOREST, said, "xFOREST Therapeutics has been expanding its proprietary RNA-targeted drug discovery technology centered on FOREST, a large-scale, small molecule discovery technology targeting RNA structures. (otsuka.co.jp)
Cognition's lead product candidate, Elayta (CT1812), a highly brain penetrant small molecule with a unique disease-modifying synaptorestorative mechanism of action, is currently in Phase 2 clinical testing for mild-to-moderate Alzheimer's disease. (pipelinereview.com)
Together, these data highlight MC3-R as a potential therapeutic target and suggest that small molecule agonists directed at MC3-R with more specific actions, may be potentially novel therapeutics for treating this pathology. (westminster.ac.uk)

At SwanBio Therapeutics, we enjoy the confidence and support of both Syncona Ltd. and Partners Innovation Fund in achieving a focused multi-program platform in neurological diseases through what has become the most significant investment in the company to date," said Tom Anderson , CEO and Director of SwanBio Therapeutics. (prnewswire.com)
We continue to seek and develop gene therapies in neurological diseases where there is significant unmet need and where our science can make a difference in the lives of patients and their families. (prnewswire.com)
SwanBio Therapeutics is a privately-held company focused on the development and commercialization of adeno-associated virus (AAV) based gene therapies for the treatment of neurological diseases with significant unmet medical need. (prnewswire.com)
The OHC prioritizes rare genetic diseases affecting children or adults where there is urgent need and opportunity for greatest impact and that fall into the areas of rare neurological diseases, cancers and developmental diseases. (ox.ac.uk)

This new collaboration gives us the opportunity to build on that momentum and expand the reach of our combined expertise to a challenging, severe rare liver disease of high unmet need. (hemoshear.com)

Chris Hollowood , Chief Investment Officer of Syncona Ltd. and Chair of SwanBio Therapeutics, said: "This financing, which is Syncona's largest Series A to date, demonstrates our belief that we have a differentiated opportunity to build a world-class company targeting neurodegenerative diseases, where there are no current treatments available. (prnewswire.com)
Both hold professorships in Physical Therapy (PT) at their respective universities, where their research focuses on the benefits of PT interventions in neurodegenerative disorders like Huntington's Disease. (hdsa.org)
The company's pipeline consists of seven clinical and preclinical neuroscience programs that target novel mechanisms of action for a broad range of underserved neuropsychiatric disorders and neurodegenerative diseases. (renaissancecapital.com)
PITTSBURG, PA, USA I November 08, 2018 I Cognition Therapeutics, Inc. , a clinical stage neuroscience company focused on synaptic protection and restoration in Alzheimer's disease and other neurodegenerative disorders, today announced that patient treatment has begun in the Phase 2 SHINE study of Elayta™ (CT1812), Cognition's lead candidate for the treatment of mild-to-moderate Alzheimer's disease. (pipelinereview.com)
Prion disease is a rapidly progressing, fatal neurodegenerative disease caused by the misfolding of the cellular prion protein, PrP C , encoded by the PRNP gene. (sangamo.com)

To analyze and study the global Biopolar Disorder Therapeutics sales, value, status (2013-2017) and forecast (2018-2025). (medgadget.com)
Sangamo expects to submit a potential IND for a product candidate treating prion disease in 2025. (sangamo.com)

Highlights of the book include: The book offers an in-depth look at more than seventy different types of skin diseases It explains in detail the clinical description, symptomatology, diagnosis, pathology, etiology, and treatment of various skin diseases. (bjainbooks.com)
The SHINE study is the culmination of substantial preclinical and clinical work characterizing Elayta and its unique synaptorestorative mechanism of action," added Cognition Chief Science Officer Susan Catalano, Ph.D. "Our clinical development program has been carefully designed to provide efficacy and safety evidence as well as important insights into the pathology of Alzheimer's disease and biomarkers of disease progress. (pipelinereview.com)

Huntington's disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. (hdsa.org)
Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam s RNAi therapeutic platform, function upstream of today s medicines by potently silencing messenger RNA (mRNA) the genetic precursors that encode for disease-causing proteins, thus preventing them from being made. (webwire.com)
This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. (webwire.com)
At Moderna, we are working to intensify this light across an array of rare genetic diseases - and our intent is to bring the full potential of our mRNA-based medicines to create new therapies for untreatable, and previously undruggable ailments. (modernatx.com)
We have long believed that our platform has great potential in treating rare genetic diseases. (modernatx.com)
The potential of our innovative medicines to treat patients with rare genetic diseases is profound. (modernatx.com)
Our scientists tap into this powerful biology for patients, and it is our belief that once we demonstrate the ability to successfully replace a missing protein in one rare genetic disease, we should be able to treat other like diseases using a similar approach. (modernatx.com)
This provides us with a unique opportunity to make a difference in not just one or two diseases, but across a vast array of rare genetic diseases in a relatively short period of time. (modernatx.com)
These diseases are caused by a genetic defect that results in either too little or no production of a critical metabolic enzyme produced by liver cells, leading to recurring, life-threatening metabolic events as the patient's blood becomes highly acidic. (modernatx.com)
We now are looking at new opportunities to use our mRNA platform to instruct the liver to direct expression of missing enzymes in patients with other genetic diseases. (modernatx.com)
With the speed at which we are able to advance programs and the ability to scale development and manufacturing to target multiple rare genetic diseases, we hope to drive improved patient outcomes - and the associated economic benefits - to a broad swath of the rare disease community. (modernatx.com)
On this Rare Disease Day, we are still in the early stages of our efforts, but we have a great deal of excitement and optimism that we can bring better outcomes to those living with rare genetic diseases. (modernatx.com)
Based on a large body of genetic, clinical, and observational evidence showing the effects of higher levels of HbF in patients with SCD, the induction of HbF by 5-10% over baseline is associated with reduced disease burden and improved clinical outcomes. (benzinga.com)
We are pleased to enter into a collaboration agreement with the world-renowned Sheba Medical Center for the development of ASO-based treatments for rare genetic diseases. (ktla.com)
7 - 9 A TMA is called "primary" when a genetic or acquired defect in a complement protein is identified (as in atypical hemolytic uremic syndrome [aHUS]) or "secondary" when occurring in the context of another disease process or factor such as infection, autoimmune disease, malignancy, or drugs. (jrheum.org)
Our unique ability to recreate models of complex rare diseases will enable Takeda to interrogate their drug targets in the genetic background of human disease and select lead candidates in an accelerated timeframe. (hemoshear.com)
We are looking for scientists that apply this emerging technology to human neurons and organoids, with the objective of discovering novel genes critical in neuronal maturation and function, and/or developing multiplexed approaches to evaluate the combinatorial interactions between the hundreds of genetic variants underlying neuropsychiatric disease risk. (sciencecareers.org)
Next generation human genome editing and forward genetic neuronal screens will allow members of CDN to resolve the interactions between genetic variants, cell types and molecular functions within an enhanced disease-relevant context. (sciencecareers.org)
Decuprate is initially being developed as a novel treatment for Wilson's disease, a rare genetic disease that affects approximately 1 in 30,000, causing copper overload in the liver, brain and other tissues and resulting in organ damage and dysfunction. (wikipedia.org)
Sickle cell disease (SCD) has its roots in genetic mutations that cause a single amino acid change in the β-globin chain of hemoglobin A and thereby confer protection against malaria. (nih.gov)
Gaucher disease is a rare genetic disorder characterized by the deposition of glucocerebroside in cells of the macrophage-monocyte system. (medscape.com)

citation needed] As of 2016, tetrathiomolybdate had been tested in over 500 patients for up to seven years, primarily in oncology and Wilson's disease, as well as some other clinical pathologies. (wikipedia.org)

Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. (newswise.com)
Its goal is to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years and target multiple approvals from regulators in key markets including the U.S., the U.K. and Europe. (ox.ac.uk)
The collaboration could potentially be expanded to cover ulcerative colitis, another inflammatory bowel disease, under certain conditions. (financialcontent.com)
TScan's platform, as per Gavin MacBeath, acting CEO and CSO at TScan, is specifically designed to identify antigens targeted by the immune system in IBD, thereby potentially enabling the development of first-in-class therapeutics to address these unmet medical needs. (financialcontent.com)
I'm excited to combine Sangamo's cutting-edge epigenetic regulation capabilities with the delivery abilities of Voyager's capsid to potentially create the first ever meaningful therapy for prion disease. (sangamo.com)

The report covers forecast and analysis for the rheumatology therapeutics market on a global and regional level. (medgadget.com)
The study includes drivers and restraints for the rheumatology therapeutics market along with the impact they have on the demand over the forecast period. (medgadget.com)
Additionally, the report includes the study of opportunities available in the rheumatology therapeutics market on a global level. (medgadget.com)
This report offers comprehensive coverage of global rheumatology therapeutics market along with, market trends, drivers, and restraints of rheumatology therapeutics market. (medgadget.com)
To understand the competitive landscape in the market, an analysis of Porters five forces model for the rheumatology therapeutics market has also been included. (medgadget.com)
The study provides a decisive view on the rheumatology therapeutics market by segmenting the market based on disease type, drug type, distribution channel, and region. (medgadget.com)
On the basis of disease type, the global rheumatology therapeutics market is divided into rheumatoid arthritis, osteoarthritis, lupus, gout, ankylosing spondylitis and other. (medgadget.com)
Rheumatoid arthritis disease has high prevalence rates than other disease and accounts for the major share in global rheumatology therapeutics market. (medgadget.com)
The distribution channel segment of rheumatology therapeutics includes a retail pharmacy, online pharmacy, and hospitals. (medgadget.com)

The complement system is a tightly regulated, cascading protein network that performs multiple roles in homeostasis and disease prevention and is a key component of both the innate and the humoral immune systems. (jrheum.org)
There are currently no approved or clinical-stage disease-modifying therapies for the prevention or treatment of prion disease. (sangamo.com)
Centers for Disease Control and Prevention. (cdc.gov)
The Centers for Disease Control and Prevention (CDC) cannot attest to the accuracy of a non-federal website. (cdc.gov)
Ongoing efforts are needed to develop more effective strategies , particularly for individuals with severe disease , for prevention and treatment of viral persistence in immune-privileged sites, for optimisation of post-exposure prophylaxis , and to increase therapeutic breadth. (bvsalud.org)
However, despite clinical trials that demonstrate the effectiveness of intrapartum antibiotic prophylaxis, prevention strategies have not been implemented widely or consistently, and the incidence of neonatal GBS disease has not declined. (cdc.gov)
This report provides the epidemiologic basis for prevention protocols, summarizes results of clinical trials demonstrating the efficacy of intrapartum antimicrobial agents, examines limitations of different approaches to prevention, and presents guidelines for the prevention of GBS disease. (cdc.gov)
and education of prenatal patients regarding GBS disease and the available prevention policy. (cdc.gov)
CDC developed these GBS disease prevention guidelines through critical analysis of clinical trial data and subsequent review of guidelines by consultants representing numerous disciplines. (cdc.gov)
I'm Commander Ibad Khan and I'm representing the Clinician Outreach and Communication Activity COCA with the Emergency Risk Communication Branch at the Centers for Disease Control and Prevention. (cdc.gov)
Thanks to the combined work of countless scientists, medical professionals, and epidemiologists, we have rapidly learned about the pathogenesis, prevention, and treatment of this disease. (cdc.gov)
The medical science concerned with the prevention, diagnosis, and treatment of diseases in animals. (bvs.br)

Lichfield-based Strolll is a medical technology company that develops digital therapeutics software for commercially available augmented reality glasses, which transforms physiotherapy and rehabilitation for people living with neurological disorders. (thebusinessdesk.com)

A new market study, titled "Global Biopolar Disorder Therapeutics Market Research Report 2018", has been featured on WiseGuyReports. (medgadget.com)

Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. (webwire.com)
From the neonate to the senior citizen, respiratory diseases can have severe acute manifestations or can involve long-term chronic symptoms, each of which can severely and adversely affect lung function. (ddw-online.com)
AVTX-002 (AEVI-002, KHK-252067,SAR-252067) is under development for the treatment of severe pediatric onset Crohn's disease, moderate to severe Crohn's disease, COVID-19 cytokine storm induced acute respiratory distress syndrome, pneumonia, acute lung injury and non-eosinophilic asthma (NEA). (pharmaceutical-technology.com)
Bone marrow transplants from healthy donors are used successfully to treat certain blood cancers for which the course of the disease is so severe that the destruction of the patient's own immune system through high-dose chemotherapy and radiation seems an effective compromise. (nih.gov)
The factors that contribute to neurologic involvement in patients with types 2 and 3 disease are still unknown but may be related to the accumulation of a cytotoxic glycolipid, glucosylsphingosine, in the brain due to the severe deficiency of glucocerebrosidase activity or to neuroinflammation. (medscape.com)
Disease having a short and relatively severe course. (bvs.br)

Takeda is deeply committed to pursuing innovative approaches that target central nervous system diseases with no available or effective treatments," said Emiliangelo Ratti, Head, Neuroscience Therapeutic Area at Takeda. (biospace.com)
HemoShear has a successful ongoing three-year collaboration with Takeda to discover and develop novel therapeutics for liver diseases, including nonalcoholic steatohepatitis (NASH). (hemoshear.com)
We are excited to expand our collaboration with Takeda into modeling rare liver diseases,' said Brian Wamhoff, PhD, chief operating officer and head of innovation, HemoShear. (hemoshear.com)
In addition to the Company's proprietary rare disease programs, HemoShear has exclusive partnerships to identify novel therapeutic approaches in nonalcoholic steatohepatitis (NASH) with Takeda and in gout with Horizon Therapeutics . (hemoshear.com)
The Milner Therapeutics Institute are excited to announce that two new pharma partners - Takeda and Sanofi - have joined the Milner Consortium in the past month. (bioindustry.org)

Using its proprietary epigenetic regulation platform, Sangamo has developed zinc finger transcriptional regulators (ZF-TRs) which it believes can specifically and potently block expression of the prion protein, the pathogenic driver of prion disease. (sangamo.com)
Wilson Therapeutics' lead product, Decuprate, is the proprietary bis-choline salt of tetrathiomolybdate. (wikipedia.org)

Dr. John Farley, the Director of the Office of Infectious Diseases at the Office of New Drugs at the Center for Drug Research at the US Food and Drug Administration. (cdc.gov)

Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy? (nih.gov)

Materia Medica of more than 180 remedies showing their specific indications for diseases of the skin. (bjainbooks.com)
The author has included a section on general anatomy and physiology as well as a Materia Medica of 185 remedies showing their specific indications for diseases of the skin. (bjainbooks.com)
Neumora Therapeutics, a Phase 3 biotech developing therapies for neuropsychiatric indications, raised $250 million by offering 14.7 million shares at $17, within the range of $16 to $18. (renaissancecapital.com)

Increase in investment in research and development and collaboration activities between companies for product development are the other factors likely to propel the bipolar disorder therapeutics market. (medgadget.com)
This collaboration aims to advance treatments for inflammatory bowel diseases (IBD), a term that encompasses both Crohn's disease and ulcerative colitis. (financialcontent.com)
Otsuka Pharmaceutical Co., Ltd. (Otsuka) announces that it has entered into a collaboration agreement with xFOREST Therapeutics Co., Ltd. (xFOREST) with the aim to create RNA structure-targeted drugs in multiple diseases. (otsuka.co.jp)
Through this collaboration on a new disease target, we will strive to be a driving force for the development of therapeutic drugs as soon as possible. (otsuka.co.jp)
NEW YORK ─ Bio-Me and Siolta Therapeutics on Monday announced that they have entered into a collaboration and marketing agreement to develop a rapid diagnostic test for newborns at risk of developing allergy and asthma later in life. (genomeweb.com)

Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. (newswise.com)
Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com . (newswise.com)
This includes biomedical engineers, bioengineers, developmental biologists and molecular and cellular biologists that apply state-of-the-art approaches for new nucleic acid delivery technologies, such as CRISPR gene and base editing tools and oligonucleotide therapeutics, and approaches for more sophisticated control of transgene expression. (sciencecareers.org)
In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms. (genomeweb.com)

Disruption of the natural equilibria between proteases and their cognate inhibitors is a common feature of inflammatory disease. (ddw-online.com)
Although protease inhibition has been standard therapy for hereditary emphysema patients for many years, it is only recent studies that have predicted a further and more broad-based role for protease inhibitors in the treatment of respiratory disease. (ddw-online.com)
T o many, the age of blockbuster protease inhibitor therapeutics was heralded by the approval of aspartyl protease inhibitors for the treatment of HIV infection. (ddw-online.com)
Shown in Table 1 are the key members of the large families of approved aspartyl protease inhibitors that comprise the anti-hypertensive and anti-HIV therapeutics (1). (ddw-online.com)

REVEAL-Tx™ combines physiological and computational models of disease to identify novel treatment approaches and select drug candidates in a human-relevant disease context. (hemoshear.com)

Alzheimer's Disease is reported to be the most common form of dementia in developed countries, with a prevalence of about 1% at the age of 65 and over 25% in people older than 85 years of age. (visiongain.com)
Currently, advances in diagnostic technologies remain moderate and the Alzheimer's Disease biomarker market is still in its infancy. (visiongain.com)
It is expected that several diagnostic tools in the future will offer an accurate assessment of whether an individual has Alzheimer's Disease, rather than one definitive diagnostic tool. (visiongain.com)
The lead analyst of the report commented the Alzheimer's disease therapeutics market has been stagnant over the last few years, with many drugs failing late stage clinical trials. (visiongain.com)
SHINE ( S ynaptic H ealth and I mprovement of N eurological Function with E layta) is a randomized, double-blind, placebo-controlled, parallel-group Phase 2 efficacy and safety study designed to enroll up to 160 adults with mild-to-moderate Alzheimer's disease (MMSE 18-26). (pipelinereview.com)
The SHINE study, if successful, has the potential to restore the Alzheimer's community's optimism for disease-modifying therapies and give hope that people currently experiencing Alzheimer's symptoms could have meaningful stabilization or improvement of cognition. (pipelinereview.com)
We look forward to sharing our findings so that the entire Alzheimer's community may benefit from our evolving understanding of this complex disease. (pipelinereview.com)
Exploiting formyl peptide receptor 2 to promote microglial resolution: a new approach to Alzheimer's disease treatment. (westminster.ac.uk)

You'll study subject-specific content in transitional medicine and therapeutics research. (ncl.ac.uk)

Focuses on the key Biopolar Disorder Therapeutics manufacturers, to study the sales, value, market share and development plans in future. (medgadget.com)
Under the terms of the agreement, Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylam s cardiometabolic disease programs vutrisiran and ALN-AGT. (webwire.com)
Over the past six months we have started to validate these beliefs with new data and have added new rare disease development candidates to our pipeline. (modernatx.com)
However, the small number of patients for each disease has hindered development of dedicated treatments. (ktla.com)
Quisovalimab is under clinical development by Avalo Therapeutics and currently in Phase II for Crohn's Disease (Regional Enteritis). (pharmaceutical-technology.com)
This news is a "[s]ignificant development in North America as the US FDA approves it's first trial to study the use of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson's disease. (parkinson.ca)
The Center for Disease Neurogenomics is a hub of neurogenomics and disease biology, towards development and enhancement of precision medicine across neuropsychiatric disorders. (sciencecareers.org)
A zoliflodacina, um novo antibiótico oral, primeiro da classe e um dos únicos tratamentos em desenvolvimento para enfrentar a crescente ameaça da gonorreia multirresistente, entrará em ensaios decisivos graças a uma colaboração entre a Parceria Global para Pesquisa & Desenvolvimento em Antibióticos (GARDP, na sigla em inglês de Global Antibiotic Research and Development Partnership ), e Entasis Therapeutics. (dndi.org)

Top News From TCT 2017: Slideshow TCT 2017 provided new insights on how to manage left main disease, more data on bioabsorbable scaffolds, and next steps in transcatheter repair. (medscape.com)

The Washington Manual , www.unboundmedicine.com/washingtonmanual/view/Washington-Manual-of-Medical-Therapeutics/602225/all/Renal_Diseases. (unboundmedicine.com)
With all three healthcare practitioners working together on case studies that are used as examples initially, a structured approach is used to evaluate the patient, his/her diseases, evaluating the prescription, side effects, adverse drug reactions, dosing if the patient is a problem patient (renal, geriatric, hyperternsive). (skills-universe.com)

Sickle cell disease (SCD) affects millions of people throughout the world and is particularly common among those whose ancestors came from sub-Saharan Africa, certain regions in South and Central America, Saudi Arabia, India and some Mediterranean countries. (newswise.com)
People with sickle cell disease have a tremendous need for therapeutic options that can reduce morbidity and mortality and improve their quality of life,' said Julie Kanter, M.D., associate professor of Hematology and Oncology and director of the Adult Sickle Cell Program at the University of Alabama at Birmingham. (benzinga.com)
This is a compelling first look at HbF protein induction in people with sickle cell disease who have been treated with FTX-6058. (benzinga.com)
The levels of HbF protein induction that we have seen thus far reinforce our belief that FTX-6058 could provide a transformational benefit to people living with sickle cell disease,' said Bryan Stuart, president and chief executive officer at Fulcrum. (benzinga.com)
The Cellular and Molecular Therapeutics Laboratory, led by Dr. John F. Tisdale, is working on multiple strategies both in the laboratory and in the clinic to cure sickle cell disease by repairing or replacing the precursor bone marrow cells that give rise to sickled red blood cells. (nih.gov)
In addition, strategies to correct the underlying mutation which causes sickle cell disease are being pursued utilizing newly developed gene editing tools, and work in the laboratory to develop methods for efficient editing of hematopoietic stem cells is underway. (nih.gov)
Dr. Tisdale and his colleagues recently completed a modified bone marrow transplant strategy for the first time in adult patients with sickle cell disease. (nih.gov)

1 - 3 Acute kidney injury is a common prominent feature of the disease, owing to the susceptibility of the glomerular circulation to endothelial damage. (jrheum.org)
however, substantial gaps remain in improving the outcomes of acute Ebola virus disease and for the survivors . (bvsalud.org)

LEXEO Therapeutics Receives Rare Pediatric Disease Designation and Orphan Drug Designation. (curefa.org)
Decuprate has been granted orphan drug designation for the treatment of Wilson's disease in both Europe and the United States. (wikipedia.org)

Structure Therapeutics Inc.'s stock climbed 34.6% following a readout of what analysts call "competitive" and "exceptional" phase Ib data at 28 days of oral. (bioworld.com)
According to GlobalData, Phase II drugs for Crohn's Disease (Regional Enteritis) have a 48% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. (pharmaceutical-technology.com)

Sangamo is currently evaluating ZF-TRs in a variety of pre-clinical programs, including Nav1.7 for the potential treatment of chronic neuropathic pain and for prion disease. (sangamo.com)

It allows patients to access rehabilitation exercise at home and maintain their disease at a lower stage for longer. (thebusinessdesk.com)
Each year, the last day of February serves as a reminder to pause and reflect on the challenges and unique needs of patients and families living with rare diseases. (modernatx.com)
Through rigorous, innovative science and our deep commitment to listening to and working with patients and patient communities, we believe we are at the forefront of a change in how medicines for rare diseases may be delivered in the future. (modernatx.com)
HemoShear's drug discovery platform, REVEAL-Tx™ , enables the Company's scientists to create best-in-class, biologically relevant human disease models to uncover the underlying mechanisms of disease, translate those discoveries into drug targets, and select candidates that may treat patients successfully. (hemoshear.com)
Immunosuppressed chronic kidney disease (CKD) patients (e.g., patients with glomerular disease, auto-immune disorders, etc. (prnewswire.com)
A new treatment option is now available for patients suffering from agitation associated with schizophrenia or bipolar I or II disorder," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. (globenewswire.com)
These upcoming milestones, along with our strong financial foundation and late-stage programs, position BioXcel Therapeutics to deliver significant value to our shareholders while helping treat millions of patients. (globenewswire.com)
Knowledge of many of the skin manifestations in the setting of cardiac diseases has become very important and is immensely helpful for proper diagnosis and treatment of patients with cardiovascular disorders. (medscape.com)
Brazzaville University Hospital, considered a fairly worrying disease by 61.7% of patients. (who.int)
: [email protected] is a wrong perception of chronic kidney failure by patients in the end stage of their kidney disease due to the general public's low level of knowledge about kidney diseases. (who.int)
Il existe une perception erronée de l'insuffisance rénale chronique chez les patients en stade terminal de leur maladie rénale dû à la faible connaissance du grand public sur les maladies rénales. (who.int)
Patients with type 1 disease commonly present with painless splenomegaly, anemia, or thrombocytopenia. (medscape.com)
Patients with type 2 disease may present prenatally, at birth or during infancy with increased tone, seizures, strabismus, and organomegaly. (medscape.com)
Patients with type 3 disease, in addition to organomegaly and bony involvement, present with neurologic involvement, most often including slowing of the horizontal saccadic eye movements. (medscape.com)
Enzyme replacement therapy (ERT) is indicated for patients with type 1 and type 3 Gaucher disease who exhibit clinical signs and symptoms of the disease, including anemia, thrombocytopenia, skeletal disease, or visceromegaly. (medscape.com)
Substrate reduction therapy (SRT) is an alternative treatment for appropriate adult patients with type 1 Gaucher disease. (medscape.com)
ERT sometimes is started in patients with type 2 GD, as often there can be a question regarding disease type and progression, and to delay may have significant impact on patient outcomes. (medscape.com)
some patients present in childhood with virtually all the complications of Gaucher disease, whereas others remain asymptomatic into the eighth decade of life. (medscape.com)
Evans M, Carrero JJ, Bellocco R, Barany P, Qureshi AR, Seeberger A, Jacobson SH, Hylander-Rössner B, Rotnitzky A, Sjölander A. Initiation of erythropoiesis-stimulating agents and outcomes: a nationwide observational cohort study in anaemic chronic kidney disease patients. (janusinfo.se)

Strolll, a digital therapeutics software developer for Parkinson's disease, has secured £2.1m of investment. (thebusinessdesk.com)
The funding will allow the company to reach several key milestones, including gathering outcomes data from ongoing studies and NHS pilots, expanding into the US market, and demonstrating the ability to apply the technology beyond Parkinson's disease to other conditions. (thebusinessdesk.com)
Huw Sparkes, junior investment manager at Midven, said: "We are thrilled to support Strolll in its mission to transform the lives of people with Parkinson's disease and relieve strains on the NHS through innovative technology. (thebusinessdesk.com)

Faculty recruitment in Genome Editing and Therapeutics at Center for Disease Neurogenomics (CDN) at the Icahn School of Medicine at Mount Sinai in New York City. (sciencecareers.org)
We are inviting applications for tenure-track faculty position (Assistant and Associate Professor level) for scientists that are working to develop the next generation of genome editing techniques and RNA and DNA-based therapeutics. (sciencecareers.org)

Avalo Therapeutics ' prescription medications comprise aciphex sprinkle, cefaclor, millipore tablets and dose packs and millipred oral solution, among others. (pharmaceutical-technology.com)

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