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).
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

Early treatment with ACE inhibitors improves cardiac outcomes of patients with Becker muscular dystrophy

Early treatment with ACE inhibitors improves cardiac outcomes of patients with Becker muscular dystrophy

New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests

A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests

Dying patients with rare diseases struggle to get experimental therapies

Dying patients with rare diseases struggle to get experimental therapies

Anti-inflammatory nanoparticles could be applied to the weakening muscles of DMD patients

Anti-inflammatory nanoparticles could be applied to the weakening muscles of DMD patients

Intranasal COVID-19 therapeutic shows promise in preclinical trials

Intranasal COVID-19 therapeutic shows promise in preclinical trials

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

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

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

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

A new hydrogel for treatment and recovery of muscle injuries

A new hydrogel for treatment and recovery of muscle injuries

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