Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

Scientists develop new stem cell line to study conversion of stem cells into muscle

To help patients with muscle disorders, scientists at The University of Texas Health Science Center at Houston (UTHealth) have engineered a new stem cell line to study the conversion of stem cells into muscle. Findings appeared in Cell Reports.

13 Dec 2018

New mouse model shows potential for rapid identification of promising muscular dystrophy therapies

A Massachusetts General Hospital research team has created a new mouse model of a common form of muscular dystrophy with the potential of rapidly distinguishing promising therapeutic drugs from those unlikely to be successful.

13 Dec 2018

Asuragen launches assay to help diagnose Steinert’s Disease

Asuragen, Inc., a molecular diagnostics company delivering easy-to-use products for complex testing in genetics and oncology, today announced the CE marking and launch of the AmplideX DM1 Dx Kit, which simplifies the analysis of repeat expansions within the DMPK gene and is intended to aid in the diagnosis of Myotonic Dystrophy Type I (DM1), also known as Steinert's Disease.

4 Dec 2018

Researchers identify molecule to fight myotubular myopathy

Myotubular myopathy is a severe genetic disease that leads to muscle paralysis from birth and results in death before two years of age. Although no treatment currently exists, researchers from the University of Geneva, Switzerland, - working in collaboration with the University of Strasbourg, France, - have identified a molecule that not only greatly reduces the progression of the disease but also boosts life expectancy in animal models by a factor of seven.

19 Nov 2018

New model of FSHD could be useful to study effectiveness of experimental therapeutics

Facioscapulohumeral muscular dystrophy is the most prevalent dominantly inherited muscular dystrophy in the world. To date, there are no pharmacologic treatments available for the more than 850,000 people affected worldwide.

17 Nov 2018

New technology based on moths and magnets could help treat genetic diseases

A new technology that relies on a moth-infecting virus and nanomagnets could be used to edit defective genes that give rise to diseases like sickle cell, muscular dystrophy and cystic fibrosis.

14 Nov 2018

Repair defect in neurons hints at possible therapy for preventing ALS

Houston Methodist researchers have discovered a repair defect in nerve cells that send messages to the brain, opening the door to a possible therapy for preventing or slowing down ALS.

1 Nov 2018

Toxic protein assemblies found to play beneficial role in muscle generation

Toxic protein assemblies, or "amyloids," long considered to be key drivers in many neuromuscular diseases, also play a beneficial role in the development of healthy muscle tissue, University of Colorado Boulder researchers have found.

1 Nov 2018

Genetic defect linked to ALS causes sugar-starved cells to overproduce lipids

A genetic defect tied to a variety of neurodegenerative diseases and mental illnesses changes how cells starved of sugar metabolize fatty compounds known as lipids, a new study led by researchers from the Johns Hopkins Bloomberg School of Public Health shows.

31 Oct 2018

Genetic testing and breakthrough therapies may transform diagnosis, care of neuromuscular disease

A new report funded by the Muscular Dystrophy Association and released by the IQVIA Institute for Human Data Science indicates that genetic testing and breakthrough therapies will transform the diagnosis and care of neuromuscular disease within the next decade.

31 Oct 2018

MDI Biological Laboratory receives $1.2 million SEPA grant to promote data literacy

The MDI Biological Laboratory will take a national lead in teaching data literacy to students and teachers though a five-year, $1.2 million SEPA grant from the National Institute of General Medical Sciences, an institute of the National Institutes of Health.

15 Oct 2018

Study highlights role of non-mutational mechanism in myotonic dystrophy, other types of arrhythmias

A team of researchers at Baylor College of Medicine reveals new insights into what can be causing life-threatening cardiac-related events in myotonic dystrophy type 1.

28 Sep 2018

Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the patient's immune system is encountering for the first time.

4 Sep 2018

Muscle dystrophy treatment in dogs with Crispr gene editing

Researchers in the United States have shown that genetically caused muscular dystrophy in dogs could be corrected using genetic editing tools. Muscular dystrophy is one of the most common fatal genetic conditions seen in children and is also seen in dogs

31 Aug 2018

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer Inc. announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004).

30 Aug 2018

New pharmacologic approach discovered to stop immune cell death

Researchers from Case Western Reserve University School of Medicine have discovered a way to stop immune cell death associated with multiple diseases, including sepsis, inflammatory bowel disease, and arthritis.

24 Aug 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Scientists discover new genetic material that may regulate regeneration

Scientists at the MDI Biological Laboratory and the University of Maine have discovered that genetic material in the cell that was previously thought to be "junk" because of its apparent lack of function likely plays a part in regulating genetic circuits responsible for regeneration in highly regenerative animals.

22 Aug 2018

New CRISPR technology may help eliminate mutated gene sequence

In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell's internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building.

17 Aug 2018