Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
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

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

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

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

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

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

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

Study reveals link between muscular dystrophy and sphingolipids

Study reveals link between muscular dystrophy and sphingolipids

Novel gene therapy for diseased muscle fiber repair

Novel gene therapy for diseased muscle fiber repair

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

New consensus-based recommendations designed to improve quality of life for DMD patients

New consensus-based recommendations designed to improve quality of life for DMD patients

First pediatric-only guidelines for the use of pacemakers and implantable defibrillators

First pediatric-only guidelines for the use of pacemakers and implantable defibrillators

Study shows long-term safety and biologic activity of golodirsen in DMD patients

Study shows long-term safety and biologic activity of golodirsen in DMD patients

Researchers illustrate the importance and value of sharing negative research results

Researchers illustrate the importance and value of sharing negative research results

Parent Project Muscular Dystrophy successfully completes the Newborn Screening Pilot project

Parent Project Muscular Dystrophy successfully completes the Newborn Screening Pilot project

Specialized cells that keep the heart beating have ability to regenerate after birth, study reveals

Specialized cells that keep the heart beating have ability to regenerate after birth, study reveals