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.

Correcting mutations in muscle stem cells using new gene-editing technology

A new gene-editing technique can be used to correct mutations in muscle stem cells, paving the way for the first potential cell therapy for genetic muscle disorders.

30 Apr 2021

Drug cocktail can effectively activate and expand muscle stem cells

Skeletal muscle – the type of muscle that attaches to bones and enables movement – is the most abundant tissue in the human body.

27 Apr 2021

C-Path opens access to D-RSC database to accelerate therapy development for DMD

Critical Path Institute announced today that it will open access to the Duchenne Regulatory Science Consortium (D-RSC) database to qualified researchers, through its Rare Disease Cures Accelerator, Data and Analytics Platform.

22 Apr 2021

Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon.

20 Apr 2021

Novel gene mutations may help identify patients with a specific form of muscular dystrophy

New research from the University of Minnesota Medical School found mutations in a novel gene that may help identify patients with a specific form of muscular dystrophy.

16 Apr 2021

Japanese–European scientists detect novel genetic mitochondrial disorder

The list of known genetic mitochondrial disorders is ever-growing, and ongoing research continues to identify new disorders in this category.

15 Apr 2021

Study may lead to new therapies for patients suffering from Duchenne muscular dystrophy

A new study, led by the University of California, Irvine (UCI), reveals how chronic inflammation promotes muscle fibrosis, which could inform the development of new therapies for patients suffering from Duchenne muscular dystrophy (DMD), a fatal muscle disease.

15 Apr 2021

Study shows Urolithin A as a potent enhancer of muscle function in mice with muscular dystrophy

Progression of Duchenne Muscle Dystrophy (DMD) can be delayed in mice by supplementing their diets with Urolithin A, according to new results reported today.

9 Apr 2021

Urolithin A shows promise to slow progression of Duchenne Muscle Dystrophy

Progression of Duchenne Muscle Dystrophy (DMD) can be delayed in mice by supplementing their diets with Urolithin A, according to new results reported today.

7 Apr 2021

A new hydrogel for treatment and recovery of muscle injuries

A team from the Universitat Politècnica de València (UPV) and the CIBER Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN) has designed and tested, at a preclinical level, a new biomaterial for the treatment and recovery of muscle injuries.

31 Mar 2021

Stem cell therapy could help restore debilitating age-related muscle loss

As the name implies, induced pluripotent stem cells can become any type of cell in our body, and scientists have evidence that when they prompt them to become muscle progenitor cells they can help restore the sometimes debilitating muscle loss that happens with age.

23 Mar 2021

COVID-19 lockdown restrictions reduced light activity related to socializing and work

A study which looked at activity levels before and during the COVID-19 pandemic has found lockdown restrictions significantly reduced light activity associated with socializing and work.

22 Mar 2021

Chemical cocktail shows promise for producing muscle stem cells

A UCLA-led research team has identified a chemical cocktail that enables the production of large numbers of muscle stem cells, which can self-renew and give rise to all types of skeletal muscle cells.

19 Mar 2021

FDA approval provides targeted treatment option for patients with rare DMD mutation

Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Exons are pieces of DNA that provide information for making proteins in a person's genome).

25 Feb 2021

Canadian Neuromuscular Disease Registry increases patient access to research, clinical trials

The Canadian Neuromuscular Disease Registry (CNDR) was launched in 2010 to increase efficient patient access to cutting-edge research and clinical trials, to increase understanding of the natural history and epidemiology of neuromuscular disease across Canada, and to facilitate research collaboration.

3 Feb 2021

After a decade of lobbying, ALS patients gain faster access to disability payments

Anita Baron first noticed something was wrong in August 2018, when she began to drool. Her dentist chalked it up to a problem with her jaw.

21 Jan 2021

CoQ10 supplement could be a valuable complementary therapeutic option for various diseases

A study led by scientists from the University of Granada (UGR) has found that a supplement of Coenzyme Q10 (CoQ10), an essential molecule for life that is synthesized in the cells of our organs and tissues and is also acquired through diet, could constitute a valuable complementary therapeutic option in the treatment of certain mitochondrial diseases, colon cancer, thyroid carcinoma, and Crohn’s disease.

18 Jan 2021

Muscular Dystrophy News and Research

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