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.
UVA researchers discover potential treatment approaches for muscle loss in myotonic dystrophy type 1

UVA researchers discover potential treatment approaches for muscle loss in myotonic dystrophy type 1

New initiative to reduce treatment delays for patients with rare diseases

New initiative to reduce treatment delays for patients with rare diseases

Intranasal COVID-19 therapeutic shows promise in preclinical trials

Intranasal COVID-19 therapeutic shows promise in preclinical trials

Novel 'CopyCatcher' detects when precise copying of genetic information takes place in cells

Novel 'CopyCatcher' detects when precise copying of genetic information takes place in cells

New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

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

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

Drug cocktail can effectively activate and expand muscle stem cells

Drug cocktail can effectively activate and expand muscle stem cells

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

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

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

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

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

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

Japanese–European scientists detect novel genetic mitochondrial disorder

Japanese–European scientists detect novel genetic mitochondrial disorder

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

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

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

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

Urolithin A shows promise to slow progression of Duchenne Muscle Dystrophy

Urolithin A shows promise to slow progression of Duchenne Muscle Dystrophy

A new hydrogel for treatment and recovery of muscle injuries

A new hydrogel for treatment and recovery of muscle injuries

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

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

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

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

Chemical cocktail shows promise for producing muscle stem cells

Chemical cocktail shows promise for producing muscle stem cells

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

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

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

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