DiseasesAnalytical, Diagnostic and Therapeutic Techniques and EquipmentDisciplines and OccupationsAnthropology, Education, Sociology and Social PhenomenaTechnology, Industry, AgricultureHumanitiesHealth Care
Orphan Drug ProductionRare DiseasesDrug ApprovalEconomics, PharmaceuticalPharmacologyLegislation, DrugUnited States Food and Drug AdministrationDrug IndustryTropical MedicinePharmacology, ClinicalEthics, PharmacyOff-Label UsePatents as Topic
Molecular PharmacologyDiseasesBritish Journal of PTherapeuticsMetabolismInteractionsClinical ResearchBiochemistryIndicationsPreclinicalDiscoveryReactive oxygenDevelopmentPreventionTabletsBiotech1997AssessmentInvestigationalProteinDiseaseToxicityEfficacyHerbalConsultingCompany'sOxidative stressRegulatoryEditorialIrvineVitroStudiesEntrepreneurResearchFoodPatientsDosagePharmaceuticalCompaniesPatentsStatus
Molecular Pharmacology1
  • Mark Namchuk PhD is a professor of the practice of biological chemistry and molecular pharmacology and the executive director of therapeutics translation at Harvard Medical School. (landmarkbio.com)
Diseases13
  • Understanding these regulatory nuances can be critical for the development of such medicinal products for rare diseases, also known as orphan medicinal products. (xtalks.com)
  • Furthermore, the presentation aims to provide the audience with an understanding of how certain legal tools and incentives have stimulated the growth of the drug products for rare diseases and what other things can be done in order to achieve a better impact. (xtalks.com)
  • Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic i nclusion b ody m yositis, and a myotrophic l ateral s clerosis. (worldwide.com)
  • Conversely, drugs that stabilize HIF are being developed for the treatment of anemia and ischemic diseases (e.g., heart attack and stroke). (aspet.org)
  • As an investigational drug for numerous congenital and acquired diseases, DCA is administered orally or parenterally, usually at doses of 10-50mg/kg per day. (srcinhibitors.com)
  • DCA has been used to treat a variety of diseases (see below) but remains an investigational drug that is, thus far, not FDA approved to treat any condition. (srcinhibitors.com)
  • Tetra and Cellvera will jointly develop an oral drug candidate to combat viral diseases by leveraging expertise and resources form both companies. (tetrabiopharma.com)
  • We try to look beyond this: Research and development process leading to such orphan drugs also provides valuable insights into the natural history of the disease investigated and may even offer insights into causes and possible therapeutic options of far more common diseases. (biovalley.ch)
  • Thomas is an internationally recognized scientist with track record in clinical research of neuromuscular and neuro-ophthalmology orphan diseases and has hands-on experience in the regulatory process of orphan disease product registration. (biovalley.ch)
  • Company develops a new class of protein interaction inhibitors to tackle drug resistance in cancer and to cure other rare diseases. (biovalley.ch)
  • Over more than two decades he has directed drug discovery efforts in numerous therapeutic areas, including infectious disease, oncology, neurodegenerative and psychiatric disorders, immune-mediated inflammatory disease, inflammatory bowel disease and orphan diseases, including cystic fibrosis. (landmarkbio.com)
  • Here, we aim to explore the potential of targeting csGPCRs to treat diseases by reviewing the current knowledge of csGPCRs expressed throughout the body and by analysing the chemical space and the drug-likeness of flavour molecules. (mdpi.com)
  • 13] is due to increased side effects, lack of curative treatment for several chronic diseases, high cost of new drugs, microbial resistance and emerging diseases Humber [14]. (biomedres.us)
British Journal of P1
Therapeutics2
  • Kamada Ltd. is focused on plasma-derived protein therapeutics for orphan indications, and has a commercial product portfolio and a late-stage product pipeline. (tetrabiopharma.com)
  • Stefano is Chief Executive Officer at AZAFAROS B.V. He is an accomplished biopharma executive with over 20 years of experience and a string of successes in launch and growth of orphan medicines and specialty therapeutics. (biovalley.ch)
Metabolism1
  • XORTX is working to advance its clinical development stage products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. (itbusinessnet.com)
Interactions1
  • Due to system maintenance, the drug interactions feature you are attempting to access is temporarily unavailable. (medscape.com)
Clinical Research3
  • Acta Scientific Pharmacology (ASPC) is an international, double- blinded peer-reviewed, highly viewed, open access journal with a good impact factor that publishes highly standard scientific information on all aspects of basic research related to the field of pharmacology and Clinical Research. (actascientific.com)
  • Internationally profound journal that Publishes articles on various disciplines of Pharmacology and Clinical Research. (actascientific.com)
  • Here, we are providing an overview of clinical research in the past 10 years evaluating the anti-infective efficacy of oral small-molecule drugs for NTD(s) for which they are neither SRA approved, nor included in current WHO strategies nor, considering the research sponsors, likely to be registered with a SRA for that NTD, if found to be effective and safe. (bvsalud.org)
Biochemistry1
Indications2
  • Devimistat is undergoing multiple clinical trials and has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the following indications: Burkitt's lymphoma, biliary tract cancer, soft tissue sarcoma, myelodysplastic syndrome (MDS) and peripheral T-cell lymphoma. (cornerstonepharma.com)
  • It has since been approved for additional orphan indications, with rheumatoid arthritis as the only FDA-approved, nonorphan indication. (bvsalud.org)
Preclinical2
  • Altasciences is an international contract research organization specializing in clinical pharmacology and bioanalysis of preclinical and clinical programs. (tetrabiopharma.com)
  • Our CRO service platforms provide everything required for drug discovery and preclinical research, including specialty cell and gene therapy (CGT) therapeutic areas, such as ophthalmology, immuno-oncology, and neuroscience. (asgct.org)
Discovery5
  • The partnership will focus on the discovery and development of novel drug regimens comprising Mpex' EPIs combined with GSK's development-stage compounds. (genengnews.com)
  • Mpex will be responsible for the discovery of EPI and the development of combination drugs through clinical proof of concept. (genengnews.com)
  • Founded in 2006, Cyagen is a global provider of genetically modified rodent models and innovative cell and gene therapy (CGT) solutions for research and development (R&D), including: disease model development, AAV discovery, drug efficacy studies, and more. (asgct.org)
  • His current research interests focus on drug discovery, in particular antivirals for the treatment of COVID-19. (landmarkbio.com)
  • Namchuk joined HMS in 2020 after a 24-year research and development career in the biotech industry, in the arenas of drug discovery, translational science and pharmaceutical development. (landmarkbio.com)
Reactive oxygen1
  • It has been postulated that stimulation of PDC and respiratory chain activity by DCA in normally glycolytic Schwann cells causes uncompensated oxidative stress from increased reactive oxygen species production. (srcinhibitors.com)
Development13
  • Orphan Drug Act - Orphan Drug Act research papers examine the act passed by Congress in order to facilitate the development and production of drugs designed to treat very rare conditions. (papermasters.com)
  • Carol has about 20 years of drug development experience gained in small, medium and large pharma companies working in Germany, the UK and France. (xtalks.com)
  • From early phase and bioanalytical science s through late phase, post-approval and real-world evidence , we provide world-class, full-service drug development services. (worldwide.com)
  • Ultimately, at least a dozen new neurotransmitters found through this process went all the way through drug development. (herbalmedicineuk.com)
  • Their research supports the commercialization of generic drugs (copies of drugs), which are less expensive than brand name drugs, and participates in the development of new drugs. (tetrabiopharma.com)
  • Altus Formulation is a Quebec based drug formulation and development company using its proprietary and patent protected drug delivery technologies to generate novel, differentiated and cost-effective new products for its partners and their patients. (tetrabiopharma.com)
  • Tetra Bio-Pharma and IntelGenx Corp. have a definitive agreement for the development and commercialization of a drug product containing the cannabinoid Dronabinol XL - PPP002 for the management of anorexia and chemotherapy-induced nausea and vomiting. (tetrabiopharma.com)
  • From its robust foundation in animal model development, to implementation of artificial intelligence (AI)-powered tools for data analysis and therapeutic discoveries, Cyagen provides one-stop solutions for accelerating basic research and new drug R&D with our unique offering of models, data, algorithms, and services. (asgct.org)
  • Dr. Grace has over 30 years CMC experience as a Senior Executive encompassing CMC from analytical development and drug substance/product development, to drug substance and product manufacturing, fill-finish, QC testing and release, packaging and labelling, and supply management and distribution. (biologicsconsulting.com)
  • Development of drugs that targets the metabolic foundation for tumor growth and simultaneously harness the immune system in hard-to-treat cancers with significant medical unmet needs. (cornerstonepharma.com)
  • Innovation in oncology drug development requires a deep well of scientific, medical and business expertise, and we have assembled an unparalleled team of directors and advisors who are working with Cornerstone to revolutionize anticancer treatment. (cornerstonepharma.com)
  • His technical expertise and experience included molecular biology, cell line development, characterization, cGMP cell banking and testing, cell culture and drug substance process development. (landmarkbio.com)
  • Our review of small-molecule anti-infective drugs recently approved by a stringent regulatory authority (SRA) or in at least Phase 2 clinical development for regulatory approval showed that this pipeline cannot deliver all new treatments needed. (bvsalud.org)
Prevention1
  • For patients receiving drugs or undergoing procedures that are known to cause hemorrhagic cystitis, prevention is essential. (medscape.com)
Tablets2
  • 5.2 Drug Dependence, Withdrawal, and Abuse In the postmarketing experience with Carisoprodol Tablets, cases of dependence, withdrawal, and abuse have been reported with prolonged use. (nih.gov)
  • Most cases of dependence withdrawal, and abuse occurred in patients who have had a history of addiction or who used Carisoprodol Tablets in combination with other drugs with abuse potential. (nih.gov)
Biotech1
  • As a dynamic life-science entrepreneur he currently serves on the Board of three biotech companies with orphan disease product pipelines and is managing partner of Viopas Venture Consulting. (biovalley.ch)
19971
  • BACKGROUND: Rituximab is a top-selling biologic that was first approved by the FDA in 1997 for a non-Hodgkin lymphoma orphan indication. (bvsalud.org)
Assessment1
  • In script rapid fire l4d2 the benefit assessment is conducted as a two-step approach encompassing i scientific assessment by the Institute for Quality and Efficiency in Health Care IQWiG and ii appraisal by the G-BA for orphan drugs, both, assessment and appraisal, are performed by the G-BA. (bazancorp.com)
Investigational1
  • Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). (worldwide.com)
Protein2
  • This keynote address will describe new drugs emerging from studies of the VHL tumor suppressor protein (pVHL), which is usually defective in clear cell renal cell carcinomas (ccRCCs). (aspet.org)
  • G protein-coupled receptors (GPCRs) belong to the largest class of drug targets. (mdpi.com)
Disease7
  • This past year saw the positive completion of important pharmacology studies, receipt of orphan drug designation from the US Food and Drug Administration ("FDA") and also potential for FDA accelerated approval with the appropriate clinical data for XORLO™, the Company's proprietary formulation of oxypurinol, for the treatment of autosomal dominant polycystic kidney disease. (itbusinessnet.com)
  • James Radke (PhD, Pharmacology) transitioned from a world-renowned scientific researcher to a medical writer and rare disease expert over 20 years ago when his daughter was born with craniosynostosis. (checkrare.com)
  • Cornerstone's lead compound, CPI-613 ® (devimistat), is designed to target the mitochondria of cancer cells in order to disrupt their energy production, cutting off the fuel for disease growth. (cornerstonepharma.com)
  • Investors are attracted to the rare disease/orphan drug sector in part because it offers certain regulatory incentives and typically has exceptional revenue growth rates. (biovalley.ch)
  • The lysosomal storage disorder (LSD) alpha-Mannosidosis is a rare genetic disease and according to the EU regulations, designated as an "orphan" disease. (europa.eu)
  • It results from damage to the bladder's transitional epithelium and blood vessels by toxins, pathogens, radiation, drugs, or disease. (medscape.com)
  • Hemorrhagic cystitis results from damage to the bladder's transitional epithelium and blood vessels by toxins, viruses, radiation, drugs (in particular, chemotherapeutic drugs), bacterial infections, or other disease processes. (medscape.com)
Toxicity1
  • When administered to NHP, MDNA19 increases the absolute number of circulating CD8 T-cells in the absence of Treg and eosinophil stimulation (the latter being a major source of IL-5 production which is responsible for triggering vascular leak syndrome and associated toxicity). (newswire.ca)
Efficacy2
  • Until then, the evaluation of the efficacy and safety of drugs was not supervised by any official health authority. (theglobalfrontier.com)
  • WHO guidelines and country policies show that drugs may be recommended for control and elimination for NTDs for which they are not SRA approved (i.e. for 'off-label' use) if efficacy and safety data for the relevant NTD are considered sufficient by WHO and country authorities. (bvsalud.org)
Herbal2
  • 11]. In most countries, use of herbal medicine continues to coexist with modern Pharmacology Ernest [12]. (biomedres.us)
  • Rhein and bisdemethoxycurcumin may be potential active components for the treatment of AP crack on the hypothesis of tissue pharmacology of herbal recipe. (bazancorp.com)
Consulting1
  • Dr. Baylor joined Biologics Consulting after 20 years with the Food and Drug Administration (FDA), most recently as the director of the Office of Vaccines Research and Review (OVRR). (biologicsconsulting.com)
Company's2
  • A portfolio of nineteen patents protects the Company's biomaterials, products, production processes and methods of use. (healthstockshub.com)
  • The Company's flagship product is GLASSIA®, the first liquid, ready-to-use, intravenous plasma-derived AAT product approved by the U.S. Food and Drug Administration. (tetrabiopharma.com)
Oxidative stress2
  • Mitochondrial dysfunction including decrease of mitochondrial membrane potential and reduced ATP production represents a common final pathway of many conditions associated with oxidative stress, for example, hypoxia, hypoglycemia, and aging. (blogspot.com)
  • Piracetam treatment at concentrations between 100 and 1000 muM improved mitochondrial membrane potential and ATP production of PC12 cells following oxidative stress induced by sodium nitroprusside (SNP) and serum deprivation. (blogspot.com)
Regulatory1
  • In addition to industry appointments, he was previously an adjunct professor in the Drug Regulatory Program at LIU. (xtalks.com)
Editorial1
  • Peter Ciszewski has worked in health care publishing and media for most of his career, from overseeing editorial and production departments to running companies. (checkrare.com)
Irvine1
  • Currently there are over 10,000 herbs in the TCM canon, according to Olivier Civelli, a Swiss-born molecular biologist at the University of California, Irvine, who, in the mid-1990s, invented the "reverse pharmacology" process now being applied to that list of plants. (herbalmedicineuk.com)
Vitro1
  • The combination of prednisolone and the antithrombotic drug dipyridamole was profiled using in vitro and in vivo models of anti-inflammatory activity and glucocorticoid-induced adverse effects to demonstrate a dissociated activity profile. (biomedcentral.com)
Studies1
  • The evaluation of the biogenic amine steady state level is currently considered a useful tool to predict the effect of a drug on the activity in the brain, particularly in in vivo studies [44,45]. (researchgate.net)
Entrepreneur1
  • He is an avid patient-centric drug developer and entrepreneur. (xeromedia.com)
Research6
  • Aids Drug Assistance Program - AIDS Drug Assistance Program Research Papers examine the programs primary purpose and discuss the CARE Act. (papermasters.com)
  • Lsd Lysergic Acid Diethylamide - LSD - Lysergic Acid Diethylamide Research Papers examine a popular hallucinogenic drug in the 1960's. (papermasters.com)
  • Pharmaceutical drug importation - Research Papers on Pharmaceutical Drug Importation and the FDA Laws. (papermasters.com)
  • Paper Masters custom writes research on the problem and impact of pharmaceutical drug importation. (papermasters.com)
  • Psychoactive Drugs - Psychoactive Drugs Research Papers explore the advantages and disadvantages of psychoactive drugs. (papermasters.com)
  • Stimulants In Sports - Stimulants in Sports research papers discuss the issue of drugs in professional and amateur sports. (papermasters.com)
Food1
  • In August 2011, Anascorp was approved by the US Food and Drug Administration (FDA) for the treatment of Centruroides exilicauda envenomations. (mhmedical.com)
Patients2
  • and the exorbitant drug prices that harm patients. (ncl.edu.tw)
  • Recently, the orphan drug palocarotene, was approved to treat patients. (checkrare.com)
Dosage1
  • Drugs can be used to treat different illnesses and improve health condition of a person, but at the same time their misuse, wrong dosage or at least minimally incorrect calculations can lead to unacceptable health risks. (actascientific.com)
Pharmaceutical1
  • The project's goal is to isolate every compound contained in all the medicinal herbs in the Chinese healing tradition, and to utilize modern pharmaceutical practices to turn these plants into more efficient-and more marketable-drugs. (herbalmedicineuk.com)
Companies1
  • Some drug companies, brand biologic companies, in particular, have been accused of covering only a single drug with more than eighty patents. (ncl.edu.tw)
Patents4
  • These drug patents accumulate to what critics claim as one of the major culprits of high drug prices- "patent thickets. (ncl.edu.tw)
  • This article aims to provide further insight regarding this issue by analyzing and comparing the U.S. patents that cover top-selling biologics and small-molecule drugs. (ncl.edu.tw)
  • Results not only confirm the existence of biologic patent thickets but also show that among the top-selling drugs, more patents cover biologics than small-molecule drugs. (ncl.edu.tw)
  • Defined as large numbers of non-overlapping or inventive patents that cover different aspects of the drug, Type I Patent Thickets are formed due to the complex nature of biologics and biosimilars. (ncl.edu.tw)
Status1
  • In June 2000, Silanes Laboratory received orphan drug status for a Centruroides scorpion antivenom, an equine-derived F(ab′) 2 from Centruroides limpidus , Centruroides noxius, Centruroides suffusus suffusus, and Centruroides meisei (formerly known as Centruroides elegans ) manufactured by Instituto Bioclon of Mexico, and referred to as Anascorp. (mhmedical.com)