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).
Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

New form of gene editing slows ALS progression in mice

New form of gene editing slows ALS progression in mice

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Simple accelerometers improve diagnosis of Duchenne muscular dystrophy

Simple accelerometers improve diagnosis of Duchenne muscular dystrophy

New gene correction therapy for hereditary muscular disease among children

New gene correction therapy for hereditary muscular disease among children

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

Horizon Discovery to offer novel base editing technology for clinical research

Horizon Discovery to offer novel base editing technology for clinical research

Researchers find key to preventing muscular dystrophy-related heart disease

Researchers find key to preventing muscular dystrophy-related heart disease

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