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.
Experimental drug prolongs survival, improves muscle function in mice severe Duchenne muscular dystrophy

Experimental drug prolongs survival, improves muscle function in mice severe Duchenne muscular dystrophy

Researchers receive $1.2 million Keck Foundation grant to find molecules for targeting the 'undruggable proteome'

Researchers receive $1.2 million Keck Foundation grant to find molecules for targeting the 'undruggable proteome'

Cancer drug helped delay progression of Duchenne muscular dystrophy in mice

Cancer drug helped delay progression of Duchenne muscular dystrophy in mice

Existing cancer drug shows promise for treating patients with muscular dystrophy

Existing cancer drug shows promise for treating patients with muscular dystrophy

NUS researchers discover a new way to interpret unsolved Mendelian diseases

NUS researchers discover a new way to interpret unsolved Mendelian diseases

U-M researchers reveal how a mutated protein acts as risk factor in Parkinson’s disease

U-M researchers reveal how a mutated protein acts as risk factor in Parkinson’s disease

The promise of RNA medicine is targeted and personalized

The promise of RNA medicine is targeted and personalized

Study supports daily steroid doses for children with Duchenne muscular dystrophy

Study supports daily steroid doses for children with Duchenne muscular dystrophy

DGP student investigates sexual dysmorphism in mice

DGP student investigates sexual dysmorphism in mice

Researchers develop new platform capable of mitochondrial DNA editing

Researchers develop new platform capable of mitochondrial DNA editing

Lab grown, self-renewing muscle stem cells can repair muscle tissue damage in mice

Lab grown, self-renewing muscle stem cells can repair muscle tissue damage in mice

Regular cycling can improve mobility in patients with myotonic dystrophy

Regular cycling can improve mobility in patients with myotonic dystrophy

Study shows the benefits of once-weekly prednisone in obese mice

Study shows the benefits of once-weekly prednisone in obese mice

Using CRISPR/Cas to develop biosafety materials

Using CRISPR/Cas to develop biosafety materials

Reactivating Piezo1 protein could help repair broken down, dystrophic muscles

Reactivating Piezo1 protein could help repair broken down, dystrophic muscles

Using mRNA to introduce gene editor into human muscle stem cells

Using mRNA to introduce gene editor into human muscle stem cells

Cellular therapy offers promise for patients with Duchenne muscular dystrophy, trial shows

Cellular therapy offers promise for patients with Duchenne muscular dystrophy, trial shows

Glucocorticoid steroids improve muscle performance through distinct, sex-specific molecular pathways

Glucocorticoid steroids improve muscle performance through distinct, sex-specific molecular pathways

First platform trial for ALS hopes to accelerate the development of effective treatments

First platform trial for ALS hopes to accelerate the development of effective treatments

Inside the tactical tug of war over the controversial Alzheimer’s drug

Inside the tactical tug of war over the controversial Alzheimer’s drug