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).
UCI researchers discover gene critical for muscle repair

UCI researchers discover gene critical for muscle repair

C-Path releases new publication on transforming drug development for neurological disorders

C-Path releases new publication on transforming drug development for neurological disorders

New drug gets FDA approval for the treatment of common genetic disease in young boys

New drug gets FDA approval for the treatment of common genetic disease in young boys

FDA approves a new steroidal-type anti-inflammatory drug for Duchenne muscular dystrophy

FDA approves a new steroidal-type anti-inflammatory drug for Duchenne muscular dystrophy

Dual CRISPR-Cas3 is a promising tool to induce a gigantic genomic deletion and restore dystrophin protein

Dual CRISPR-Cas3 is a promising tool to induce a gigantic genomic deletion and restore dystrophin protein

Next-generation sequencing provides novel insights into the mechanisms underlying autism and myotonic muscular dystrophy

Next-generation sequencing provides novel insights into the mechanisms underlying autism and myotonic muscular dystrophy

Duchenne and other dystrophinopathies research receives major boost from new collaboration

Duchenne and other dystrophinopathies research receives major boost from new collaboration

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

New porcine model could help develop treatments for Dduchenne muscular dystrophy

New porcine model could help develop treatments for Dduchenne muscular dystrophy

Alternative splicing in neurodegenerative disease and the promise of RNA-based therapeutic strategies

Alternative splicing in neurodegenerative disease and the promise of RNA-based therapeutic strategies

Manipulating a signaling protein to slow down muscular dystrophy

Manipulating a signaling protein to slow down muscular dystrophy

Scarring to the collagen framework causes dysfunction in Duchenne muscular dystrophy

Scarring to the collagen framework causes dysfunction in Duchenne muscular dystrophy

Novel molecule may help overcome the devastating symptoms of Duchenne muscular dystrophy

Novel molecule may help overcome the devastating symptoms of Duchenne muscular dystrophy

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

Using motion capture technology and AI to monitor the progression of movement disorders

Using motion capture technology and AI to monitor the progression of movement disorders

Gene editing could offer a promising solution for treating patients after a heart attack

Gene editing could offer a promising solution for treating patients after a heart attack

Mutations responsible for common inherited heart condition corrected using CRISPR-Cas9 therapy

Mutations responsible for common inherited heart condition corrected using CRISPR-Cas9 therapy

Researchers receive $9 million grant to study how ‘jumping genes’ influence Alzheimer's disease

Researchers receive $9 million grant to study how ‘jumping genes’ influence Alzheimer's disease

New discovery changes the understanding of Duchenne muscular dystrophy

New discovery changes the understanding of Duchenne muscular dystrophy