Type of muscular dystrophy myotonic dystrophy. Medical search. Frequent questions

Congenital muscular dystrophies, which are present at birth or before age 2. (medlineplus.gov)
Weakness in respiratory muscles can cause difficulties with breathing, and heart rhythm abnormalities can lead to repeated bouts of fatigue, dizziness, and fainting.Rarely, infants can have this form of MD, known as congenital myotonic dystrophy. (drweil.com)
This is for individuals who have been diagnosed with Bethlem myopathy, Ullrich congenital muscular dystrophy, or an intermediate form of these conditions. (musculardystrophyuk.org)
Muscular Dystrophy UK has formed a partnership with the American patient organisation CureCMD to support the international congenital muscular dystrophy registry (CMDIR). (musculardystrophyuk.org)
However, FKRP mutations may also cause a form of congenital muscular dystrophy (CMD) known as MDC1C, and in rare instances, the diseases known as Muscle Eye Brain (MEB) disease and Walker-Warburg Syndrome (WWS). (musculardystrophyuk.org)
Methods We analysed 104 in-laboratory sleep studies of 73 patients with MD with five common types (DMD-Duchenne, Becker MD, CMD-congenital, LGMD-limb-girdle and DM-myotonic dystrophy). (bmj.com)
This study looks into five major types of MD (Duchenne MD, Becker MD, congenital MD, LGMD-limb-girdle and DM-myotonic dystrophy). (bmj.com)
Fukuyama congenital muscular dystrophy (FCMD) is the second most common muscular dystrophy in Japan, usually due to a founder mutation of the fukutin gene. (myobase.org)
In May 2022, In response to developments in AMO Pharma's potentially crucial REACH-CDM study, a double-blind, placebo-controlled, randomised clinical trial to evaluate the efficacy and safety of AMO-02 (tideglusib) for the treatment of congenital myotonic dystrophy (CDM1), the company AMO Pharma Limited announced that its current investors have increased their investments in the business. (abnewswire.com)
Infants with congenital myotonic dystrophy have severely reduced muscle tone (hypotonia, or "floppiness"), feeding and breathing problems, bone deformities, facial weakness, and delays in development of thought processes and physical movement. (msdmanuals.com)
Congenital muscular dystrophies progress slowly and affect males and females. (healthnbeautytips.co)
The two forms that have been identified - Fukuyama and congenital muscular dystrophy with myosin deficiency - cause muscle weakness at birth or in the first few months of life, along with severe and early contractures. (healthnbeautytips.co)
Tent-shaped mouth as a presenting symptom of congenital myotonic dystrophy. (symptoma.com)
Genetic epidemiology of congenital muscular dystrophy in a sample from north-east Italy. (symptoma.com)

June 21, 2023 - Notice of Intent to Publish a Funding Opportunity Announcement for Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Centers (MDSRC) (P50 Clinical Trial Optional). (nih.gov)
WALTHAM, Mass., Sept. 20, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). (tmcnet.com)
" Myotonic Dystrophy Pipeline Insight, 2023 " report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Myotonic Dystrophy Market. (abnewswire.com)
In February 2023, Entrada announced the closing of its strategic collaboration and license agreement with Vertex for the discovery and development of intracellular EEV-therapeutic candidates for myotonic dystrophy type 1 (DM1). (biospace.com)
In March 2023, Entrada presented additional preclinical data at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference supporting the development of ENTR-601-45 for the potential treatment of patients with Duchenne who are exon 45 skipping amenable, including data visualizing dystrophin restoration in skeletal and cardiac muscle cells. (biospace.com)
2023, Muscular Dystrophy Association Inc. All rights reserved. (mda.org)

Becker muscular dystrophy, which is similar to Duchenne but is less severe and gets worse more slowly. (medlineplus.gov)
National registries have been developed for Duchenne and Becker muscular dystrophies. (musculardystrophyuk.org)
People with Duchenne or Becker muscular dystrophy can register with the UK DMD Registry which is managed by Action Duchenne. (musculardystrophyuk.org)
Like Duchenne muscular dystrophy, Becker muscular dystrophy typically affects only males (1 in 30,000) and causes heart problems. (healthnbeautytips.co)
We identified sources of variation in prevalence estimates of Duchenne and Becker muscular dystrophy (DBMD) within MD STARnet from published literature and a survey of MD STARnet investigators, then developed a logic model of the relationships between the sources of variation and estimated prevalence. (cdc.gov)

What are the symptoms and forms of muscular dystrophy? (drweil.com)
Myotonic muscular dystrophy is one of the most common forms of muscular dystrophy. (stanfordchildrens.org)
It is generally less severe, progresses more slowly, and affects fewer muscles than other forms of muscular dystrophy. (healthnbeautytips.co)

Facioscapulohumeral muscular dystrophy, which often starts in the teenage years. (medlineplus.gov)
Dyne is advancing treatments for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). (dyne-tx.com)

The company announced new non-human primate (NHP) data demonstrating durability of response through 12 weeks for ENTR-601-44, an EEV-conjugated phosphorodiamidate morpholino oligomer (PMO) for the potential treatment of people with Duchenne muscular dystrophy (DMD) who are exon 44 skipping amenable. (parentprojectmd.org)

In some types of neuromuscular disease, problems in the digestive tract, central nervous system, or autonomic nervous system may also contribute to sleep issues. (bellaonline.com)
Sleep apnea often affects individuals with other neuromuscular diseases, such as type 1 myotonic muscular dystrophy and Duchenne muscular dystrophy, as well. (bellaonline.com)
Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and spinal muscular atrophy (SMA) are the most prevalent neuromuscular disorders (NMDs) in children and adults. (mcmaster.ca)
Every day Muscular Dystrophy UK is working towards a future with effective treatments and ultimately cures for all muscle-wasting and associated neuromuscular conditions. (musculardystrophyuk.org)
The team at the Stanford Medicine Children's Health Pediatric Neuromuscular Diseases Clinic works together to develop the best possible care plan for a child with Duchenne muscular dystrophy. (stanfordchildrens.org)
Muscle fatigue can be central, neuromuscular, or peripheral muscular. (rxharun.com)

Metformin (N,N-dimethyl-biguanide) for Steinert myotonic dystrophy. (afm-telethon.fr)
Myotonic dystrophy type I (DM1), also known as Steinert disease, and myotonic dystrophy type II (DM2), also known as proximal myotonic myopathy, which is a milder form of DMI, are the two main forms recognised based on clinical and molecular presentation. (abnewswire.com)
There are two major forms recognized based on clinical and molecular presentation: Myotonic dystrophy type I (DM1), known as Steinert disease, and myotonic dystrophy type II (DM2), or proximal myotonic myopathy which is a milder variety of DMI. (giridihjournal.in)

This rare form of muscular dystrophy appears from childhood to early teens and affects mainly males. (healthnbeautytips.co)
PepGen can start a Phase 1 study in the U.S. of a therapy for a rare form of muscular dystrophy after the Food and Drug Administration lifted a clinical hold on the program. (biopharmadive.com)

Both limb girdle muscular dystrophy type 2B (LGMD2B) and miyoshi myopathy are caused by mutations in a gene called dysferlin (DYSF). (musculardystrophyuk.org)
The most prevalent form of muscular dystrophy that manifests in adulthood is myotonic dystrophy (DM), which is regarded as a subtype of myopathy. (abnewswire.com)
True muscle weakness is a primary symptom of a variety of skeletal muscle diseases, including muscular dystrophy and inflammatory myopathy. (rxharun.com)
Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood. (giridihjournal.in)

Myotonic dystrophy affects about 1 in 2,100 people, a number that was long estimated to be much lower (often cited as 1 in 8,000), reflecting that not all patients have immediate symptoms and, once they do have symptoms, the long time it typically takes to get to the right diagnosis. (wikipedia.org)
Schwartz said this was "due to myotonic dystrophy type II," an inherited muscular dystrophy that affects the muscles and other body systems, such as the heart. (politifact.com)
Duchenne muscular dystrophy (DMD) is one of the most common types of muscular dystrophy, and it usually affects boys starting at about three to four years of age. (stanfordchildrens.org)
It affects what most people think of as muscle, the type that can be contracted to move the various. (msdmanuals.com)
This dystrophy affects males and females and about 1 in 8,000 people. (msdmanuals.com)
The most common form of muscular dystrophy in adults, myotonic muscular dystrophy affects both men and women, and it usually appears at any time from early childhood to adulthood. (healthnbeautytips.co)
The most common form of muscular dystrophy in children, Duchenne muscular dystrophy typically affects only males. (healthnbeautytips.co)
This form of muscular dystrophy appears in teens to early adulthood and affects males and females. (healthnbeautytips.co)
Detailed information on myotonic muscular dystrophy, a common form of muscular disease that affects adults and children. (medindia.net)
It is a rare condition with three different types, of which two affects about 1 in 8,000 people and the third only 1 in 100,000. (healthify.nz)

Clinical relevance of atrial fibrillation/flutter, stroke, pacemaker implant, and heart failure in Emery-Dreifuss muscular dystrophy: a long-term longitudinal study. (medscape.com)

Clearance of defective muscle stem cells by senolytics restores myogenesis in myotonic dystrophy type 1 (2022). (chusj.org)
13th International Myotonic dystrophy conference (2022). (chusj.org)
In July 2022, The New Zealand Medicines and Medical Devices Safety Authority approved Dyne Therapeutics, Inc.'s clinical trial application to begin its Phase I/II multiple ascending doses (MAD) clinical trials of DYNE-101 in patients with myotonic dystrophy type 1 (DM1), according to the company. (abnewswire.com)

Even within the same type of MD, people can have different symptoms. (medlineplus.gov)
Signs and symptoms vary according to the type of muscular dystrophy. (drweil.com)
Symptoms of muscular dystrophy include clumsy movement, frequent trips and falls, difficult climbing stairs, an inability to jump or hop, leg pain, facial weakness, shoulder or arm weakness, and an inability to close one's eyes or whistle. (stanfordchildrens.org)
Symptoms of myotonic dystrophy begin during adolescence or young adulthood. (msdmanuals.com)
Symptoms of myotonic dystrophy begin during adolescence or young adulthood and can range from mild to severe. (msdmanuals.com)
This form is similar to Duchenne muscular dystrophy, but the disease is much milder: symptoms appear later and progress more slowly. (healthnbeautytips.co)
An individuals age when the symptoms cropped up, the intensity with which the dystrophy spreads and the pathway (genealogy) through which the genes were forwarded are taken into consideration when diagnosing the form or type of this disorder. (healthhearty.com)
The symptoms will differ depending upon the type of dystrophy the individual is inflicted with. (healthhearty.com)
These types of MD vary in terms of when symptoms are most likely to emerge, which muscles they affect, how severe the symptoms are and whether they affect your life expectancy. (healthify.nz)

Zolgensma® (onasemnogene abeparvovec: first gene therapy treatment derived partly from research conducted at Genethon ) for spinal muscular atrophy linked to SMN1. (afm-telethon.fr)

Mothers with myotonic dystrophy may have children with a severe form of myotonia that appears during infancy. (msdmanuals.com)
However, the most severe type of genetic discrepancy is Duchenne's muscular dystrophy. (healthhearty.com)

It is the most common form of muscular dystrophy that begins in adulthood. (wikipedia.org)

The preclinical development of a gene therapy by another type of candidate drug (morpholino antisense oligonucleotides) for the exon 53 skipping enable the launch of a phase I/II human clinical trial, the SRP-4053 trial (Skip-NMD) whose results have been published. (afm-telethon.fr)

Myotonic dystrophy (DM) is a type of muscular dystrophy, a group of genetic disorders that cause progressive muscle loss and weakness. (wikipedia.org)
Duchenne muscular dystrophy is a rare, genetic disease that causes progressive muscle degeneration and weakness throughout the body. (parentprojectmd.org)
Myotonic dystrophy is an inherited disorder, the most common form of a group of conditions called muscular dystrophies that involve progressive muscle wasting and weakness. (scripps.edu)
The muscular dystrophies (MD) are a group of genetic diseases characterized by progressive weakness and degeneration in the voluntary muscles that control movement. (drweil.com)
In its most common form, Limb-girdle muscular dystrophy causes progressive weakness that begins in the hips and moves to the shoulders, arms, and legs. (healthnbeautytips.co)
DM1 is the most common muscular dystrophy in adults, and one of several genetic conditions that cause muscle wasting and progressive weakness. (medindia.net)
Muscular Dystrophy (MD) is a group of genetic discrepancies that results in weakness of the muscles further leading to a successive progression of the weakness that is caused in the muscles. (healthhearty.com)
Depending on the exact type of faulty gene and faulty protein, different types of muscle weakness result. (healthify.nz)
Although it is a commonly held belief that carriers merely pass on the disease and are unaffected, female carriers can have similar muscular weakness as affected males. (healthify.nz)
Limb-girdle muscular dystrophy refers to a group of genetic disorders that cause progressive weakness and wasting of the skeletal muscles, predominantly around the shoulders and hips. (medscape.com)

There are different gene changes that cause each type of MD. And sometimes people who have the same type of MD can have different gene changes. (medlineplus.gov)
Gene therapy trial to assess a microdystrophin (abbreviated version of the Duchenne muscular dystrophy gene) associated with an AVV vector. (afm-telethon.fr)
Mutation of the DMPK gene causes myotonic dystrophy type 1 (DM1). (wikipedia.org)
Mutation of CNBP gene causes type 2 (DM2). (wikipedia.org)
Most patients with an FKRP gene mutation have a form of limb girdle muscular dystrophy known as type R9 (LGMDR9, formerly called LGMD2i). (musculardystrophyuk.org)
Limb girdle muscular dystrophy type 2A (LGMD2A) is caused by mutations in a gene called calpain-3 (CAPN3). (musculardystrophyuk.org)
DM1 (but not Duchennes muscular dystrophy) results from a genetic error that causes expansion of a region of a particular gene, called DMPK. (illinois.edu)
Muscular dystrophy is caused by defects in certain genes, with the type determined by the abnormal gene. (healthnbeautytips.co)
In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne muscular dystrophy. (healthnbeautytips.co)
Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. (healthnbeautytips.co)

Respiratory failure is a feature of many types of LGMD. (symptoma.com)
Muscle biopsy and genetic testing are the most important tools used in the diagnostic evaluation of patients in whom limb-girdle muscular dystrophy (LGMD) is suspected. (medscape.com)
Walton and Nattrass first proposed limb-girdle muscular dystrophy (LGMD) as a nosological entity in 1954. (medscape.com)
As of 2017, there are 34 types of LGMD detailed in the OMIM database. (medscape.com)

Also, advances in therapy and therapeutical trials are presented in Duchenne and other muscular dystrophies, and some novel treatments in glycogen storage disease by enzyme replacement are proposed. (novapublishers.com)
Myotonic dystrophy (DM) is a genetic condition that is inherited in an autosomal dominant pattern, meaning each child of an affected individual has a 50% chance of inheriting the disease. (wikipedia.org)
The outlook for people with MD varies depending on the type and severity of the disease. (drweil.com)
Each form of muscular dystrophy is caused by a genetic mutation that's particular to that type of the disease. (drweil.com)
Gilbert Gottfried died from a heart problem caused by an inherited muscular dystrophy disease. (politifact.com)
The Collagen VI Alliance , which is made up of Muscular Dystrophy UK, Cure CMD, Muscular Dystrophy Ireland, AFM Telethon and Swiss Foundation for Research on Muscle Disease, is funding the development of a global registry for collagen VI-related dystrophies. (musculardystrophyuk.org)
Muscular dystrophy is a genetic disease that causes muscles to become weak and break down over time. (stanfordchildrens.org)
Myotonic (also called MMD or Steinert's disease). (healthnbeautytips.co)
Adding exercise to genetic treatment for myotonic dystrophy type 1 (DM1) was more effective in reversing fatigue than giving the treatment alone, as per the study in a mouse model of the disease. (medindia.net)
Clinical predictors of conduction disease progression in type I myotonic muscular dystrophy. (medscape.com)
Repositorio Salud Andalucía: Preimplantation Genetic Diagnosis for Myotonic Dystrophy Type 1 and Analysis of the Effect of the Disease on the Reproductive Outcome of the Affected Female Patients. (repositoriosalud.es)
ATX-01 is under development for the treatment of Myotonic Dystrophy Type 1 (DM1), a serious, debilitating, and life-threatening disease with no known cure or currently available drug treatment. (giridihjournal.in)
Enzyme replacement therapy (ERT) appears safe and effective for peripheral manifestations in patients with Gaucher disease types I and III, Fabry disease, mucopolysaccharidosis I (Hurler, Hurler-Scheie, and Scheie syndromes), mucopolysaccharidosis II (Hunter syndrome), mucopolysaccharidosis VI (Maroteaux-Lamy syndrome), and Pompe disease. (medscape.com)

Muscular dystrophy (MD) is a group of more than 30 genetic diseases . (medlineplus.gov)
Muscular Dystrophies include a heterogeneous series of diseases that range from childhood to adult onset cases of difficult diagnosis and treatment. (novapublishers.com)
Muscular dystrophy is a general term for a group of inherited diseases involving defects in the genes responsible for normal muscle functioning. (drweil.com)
His work led to the discovery of new rare myopathies, to a better understanding of the pathogenesis of muscular diseases and to the identification of new promising therapeutic approaches. (chusj.org)
Muscular dystrophy (MD) is a group of inherited diseases in which the muscles that control movement (called voluntary muscles) progressively weaken. (healthnbeautytips.co)
This study, carried out by researchers at the Massachusetts General Hospital (MGH) and collaborators, has implications for patients who experience fatigue due to genetics-related musculoskeletal diseases as well as other types of illness-induced fatigue. (medindia.net)

Nerves control the contraction of muscles by determining the number, sequence, and force of muscular contraction. (rxharun.com)

What are the treatments for muscular dystrophy (MD)? (medlineplus.gov)

Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Myotonic Dystrophy pipeline constitutes 20+ key companies continuously working towards developing 22+ Myotonic Dystrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight. (abnewswire.com)
Companies across the globe are diligently working toward developing novel Myotonic Dystrophy treatment therapies with a considerable amount of success over the years. (abnewswire.com)

The most common types of muscular dystrophy, like Duchenne's and Becker's appear to be due to a genetic mutation resulting in the absence or deficiency of dystrophin , a protein involved in maintaining the integrity of muscle. (drweil.com)
They are Myotonic dystrophy, Becker's muscular dystrophy and Limb-girdle muscular dystrophy. (healthhearty.com)

This drug, used in oncology for almost 40 years, is assessed in Duchenne muscular dystrophy, following preclinical studies supported by the AFM-Telethon. (afm-telethon.fr)

Background The characteristics of and relationship between sleep apnoea and hypoventilation in patients with muscular dystrophy (MD) remain to be fully understood. (bmj.com)
Results Patients in all five types had high risk of sleep apnoea with 53 of the 73 patients (73%) meeting the diagnostic criteria in at least one study. (bmj.com)
Conclusion Sleep-disordered breathing is common in patients with MD but each type has its unique features. (bmj.com)
however, the characteristics of sleep-disordered breathing among patients with the five types are less known. (bmj.com)
The oligonucleotide-based therapy is designed to treat patients with myotonic dystrophy type 1, or DM1. (biopharmadive.com)
Exercise plus the antisense treatment provides more benefit for patients with myotonic dystrophy. (medindia.net)

For people living with Duchenne muscular dystrophy, DMD Hub Clinical Trial Finder provides accurate and up to date information regarding clinical trials in the UK. (musculardystrophyuk.org)

Myotonic dystrophies (DM) type 1 and type 2 are the most common muscular dystrophy in adults. (igbmc.fr)

His research program aims to determine how muscle stem cell function is affected in different muscular disorders with the goal of identifying novel therapeutic avenues. (chusj.org)
Dominant x-linked disorders Genes are segments of deoxyribonucleic acid (DNA) that contain the code for a specific protein that functions in one or more types of cells in the body or code for functional RNA molecules. (msdmanuals.com)

Myotonic dystrophy type 1 (DM1) is the most common adult muscular dystrophy and presents an autosomal dominant inheritance. (repositoriosalud.es)

Myotonic Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. (giridihjournal.in)

For the first time, scientists from the Florida campus of The Scripps Research Institute (TSRI) have identified small molecules that allow for complete control over a genetic defect responsible for the most common adult onset form of muscular dystrophy. (scripps.edu)
Steinert's (Myotonic) MD is the most common adult form and is typified by prolonged muscle spasms and the inability to voluntarily relax them. (drweil.com)

The UK FSHD registry is funded by Muscular Dystrophy UK and was launched in May 2013. (musculardystrophyuk.org)

The purpose of this Funding Opportunity Announcement (FOA) is to publicize a competition for Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Centers (MDSRCs). (nih.gov)
These Centers promote collaborative basic, translational, and clinical research and provide important resources that can be used by the national muscular dystrophy research communities. (nih.gov)
The Centers also provide outstanding environments for the training of new researchers capable of addressing high priority objectives in muscular dystrophy research. (nih.gov)
A goal of this Centers program is to support important and innovative research in the muscular dystrophies that is best pursued through this interdisciplinary and collaborative center environment, and projects that may not be as effective if supported by "stand-alone" research project grants. (nih.gov)
The Centers also provide outstanding environments for the training of new scientists electing to pursue careers conducting research in high priority areas of muscular dystrophy. (nih.gov)

Duchenne muscular dystrophy, which is the most common childhood form. (medlineplus.gov)
CHAMPAIGN, Ill. - Efforts to treat myotonic dystrophy type 1, the most common form of muscular dystrophy, are in their infancy. (illinois.edu)
Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. (healthnbeautytips.co)
This form of muscular dystrophy appears in men and women in their 40s, 50s, and 60s. (healthnbeautytips.co)
In the first quarter, we successfully closed on our transformational collaboration with Vertex for the development of EEV-therapeutics targeting myotonic dystrophy type 1, the most prevalent form of muscular dystrophy. (biospace.com)
This condition can occur with no known family history of BMD or DMD, so all females who are suspected of having any form of muscular dystrophy should be tested to determine if they could be a manifesting carrier because of the genetic implications. (healthify.nz)

Namuscla® (mexiletine) for myotonic syndromes (dystrophic and non-dystrophic). (afm-telethon.fr)

Duchenne muscular dystrophy, Myotonic Dystrophy type 1) and acquired myopathies (e.g., prematurity) on muscle stem cell function, and 4) investigating new therapeutic avenues targeting defective muscle stem cells in various muscular dystrophies. (chusj.org)

Myotonic dystrophy is caused by a genetic mutation in one of two genes. (wikipedia.org)

The first authors of the study, "Induction and Reversal of Myotonic Dystrophy Type 1 Pre-mRNA Splicing Defects by Small Molecules," are Jessica L. Childs-Disney of TSRI, Ewa Stepniak-Konieczna of Adam Mickiewicz University (Poland) and Tuan Tran of TSRI. (scripps.edu)
He did his post-doctoral training at the Ottawa Hospital Research Institute in Dr. Michael Rudnicki's lab, where he studied muscle stem cell defects in Duchenne Muscular Dystrophy. (chusj.org)
Muscular dystrophy is caused predominantly by genetic defects. (healthhearty.com)
The many types of CMT are distinguished by age of onset, inheritance pattern, severity, and whether they are linked to defects in axon or myelin. (mda.org)
While these distinctions are useful, it is important to realize that because of the vast number of genetic defects that can lead to CMT, some people fall on the borders between different types, and many people have specific "subtypes" not detailed here. (mda.org)

Entrada also announced its second clinical candidate, ENTR-701, an EEV-PMO that the company is developing as a potential allele-specific treatment for people living with myotonic dystrophy type 1 (DM1). (parentprojectmd.org)
Targeting muscle stem cells with bioactive lipids for the treatment of muscular dystrophies. (chusj.org)
Myotonic Dystrophy Type 1 (DM1), a terrible, crippling, and potentially fatal condition for which there is neither a known cure nor a drug treatment in development, is the target of ATX-01. (abnewswire.com)

In non-human primates, DYNE-101 demonstrated a favorable safety profile and achieved enhanced muscle distribution as evidenced by significant reduction in wild-type DMPK RNA. (tmcnet.com)
Senolytics target defective muscle stem cells and restore myogenesis in myotonic dystrophy type 1. (chusj.org)
and muscle wasting (dystrophy) of varying severity. (msdmanuals.com)

We are proud to present new data at TIDES USA for our lead clinical candidate, ENTR-601-44 for Duchenne muscular dystrophy, and to announce our clinical candidate for myotonic dystrophy type 1 as we continue to expand our pipeline of EEV therapeutics," said Natarajan Sethuraman, PhD, Chief Scientific Officer of Entrada. (parentprojectmd.org)
The Myotonic Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. (abnewswire.com)

While myotonic dystrophy can occur at any age, onset is typically in the 20s and 30s. (wikipedia.org)

Short discrete ramps or bursts of atrial stimuli are the most likely to produce a type I conversion of atrial flutter (immediate conversion to sinus rhythm), particularly if they can be delivered in or near the flutter circuit. (medscape.com)

Anatomy10

Organisms2

Diseases27

Chemicals and Drugs26

Analytical, Diagnostic and Therapeutic Techniques and Equipment13

Phenomena and Processes25

Disciplines and Occupations1

Information Science2

Health Care3

Congenital14

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Becker5

Forms of muscular dystrophy3

Facioscapulohumeral2

People with Duchenne muscular1

Neuromuscular6

Steinert3

Rare form of muscular dystrophy2

Myopathy4

Affects10

Emery-Dreifuss1

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Symptoms9

Spinal muscula1

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Weakness10

Gene10

LGMD4

Disease13

Diseases6

Muscles1

Treatments1

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Adults1

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FSHD1

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Mexiletine1

Myopathies1

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Treatment3

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Atrial flutter1