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).
Experimental treatment shows early promise to help nearly half of DMD patients

Experimental treatment shows early promise to help nearly half of DMD patients

Cheaper drug equally effective in preserving cardiovascular function in boys with DMD

Cheaper drug equally effective in preserving cardiovascular function in boys with DMD

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Nanoparticles used to deliver CRISPR gene editing tools into the cell

Nanoparticles used to deliver CRISPR gene editing tools into the cell

New drug provides hope for patients with Duchenne muscular dystrophy

New drug provides hope for patients with Duchenne muscular dystrophy

New discovery could improve therapies for Duchenne muscular dystrophy

New discovery could improve therapies for Duchenne muscular dystrophy

Study raises the prospect of new therapies to delay muscle atrophy

Study raises the prospect of new therapies to delay muscle atrophy

Study discovers origin of neurofibromas that develop throughout the skin of NF1 patients

Study discovers origin of neurofibromas that develop throughout the skin of NF1 patients

Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Single CRISPR treatment can safely and stably correct genetic disease

Single CRISPR treatment can safely and stably correct genetic disease

Study findings hold promise for children with DMD

Study findings hold promise for children with DMD

Scientists develop new gene therapy that prevents axon destruction in mice

Scientists develop new gene therapy that prevents axon destruction in mice

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

Scientists develop new stem cell line to study conversion of stem cells into muscle

Scientists develop new stem cell line to study conversion of stem cells into muscle

Researchers identify molecule to fight myotubular myopathy

Researchers identify molecule to fight myotubular myopathy