Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.
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

New compound blocks genetic cause of familial ALS and frontotemporal dementia

New compound blocks genetic cause of familial ALS and frontotemporal dementia

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

New mouse model shows potential for rapid identification of promising muscular dystrophy therapies

New mouse model shows potential for rapid identification of promising muscular dystrophy therapies

Asuragen launches assay to help diagnose Steinert’s Disease

Asuragen launches assay to help diagnose Steinert’s Disease

Researchers identify molecule to fight myotubular myopathy

Researchers identify molecule to fight myotubular myopathy

New model of FSHD could be useful to study effectiveness of experimental therapeutics

New model of FSHD could be useful to study effectiveness of experimental therapeutics

New technology based on moths and magnets could help treat genetic diseases

New technology based on moths and magnets could help treat genetic diseases

Repair defect in neurons hints at possible therapy for preventing ALS

Repair defect in neurons hints at possible therapy for preventing ALS

Toxic protein assemblies found to play beneficial role in muscle generation

Toxic protein assemblies found to play beneficial role in muscle generation

Genetic defect linked to ALS causes sugar-starved cells to overproduce lipids

Genetic defect linked to ALS causes sugar-starved cells to overproduce lipids

Genetic testing and breakthrough therapies may transform diagnosis, care of neuromuscular disease

Genetic testing and breakthrough therapies may transform diagnosis, care of neuromuscular disease

MDI Biological Laboratory receives $1.2 million SEPA grant to promote data literacy

MDI Biological Laboratory receives $1.2 million SEPA grant to promote data literacy

Study highlights role of non-mutational mechanism in myotonic dystrophy, other types of arrhythmias

Study highlights role of non-mutational mechanism in myotonic dystrophy, other types of arrhythmias

Urine exRNA may be source of biomarkers for muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Muscle dystrophy treatment in dogs with Crispr gene editing

Muscle dystrophy treatment in dogs with Crispr gene editing

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy