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.
MDI Biological Laboratory receives $30,000 to support course in organoid technology

MDI Biological Laboratory receives $30,000 to support course in organoid technology

Discovery of small-molecule inhibitors could enable precise control over CRISPR-Cas9 genome editing

Discovery of small-molecule inhibitors could enable precise control over CRISPR-Cas9 genome editing

Reviving the heart's regenerative capacities using microRNAs

Reviving the heart's regenerative capacities using microRNAs

Research uncovers critical RNA processing aberrations in ALS and FTD

Research uncovers critical RNA processing aberrations in ALS and FTD

Scientists investigate processes that lead to neuronal death in ALS patients

Scientists investigate processes that lead to neuronal death in ALS 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

New strategy for editing and repairing genetic mutation associated with microduplications

New strategy for editing and repairing genetic mutation associated with microduplications

Bringing Maine researchers together to identify solutions to opioid epidemic in Maine

Bringing Maine researchers together to identify solutions to opioid epidemic in Maine

First-of-its-kind human trial of new brain reading device to help speech and movement

First-of-its-kind human trial of new brain reading device to help speech and movement

Potential drug targets cause of adult-onset muscular dystrophy

Potential drug targets cause of adult-onset muscular dystrophy

Gene editing and designer babies experiments face global moratorium

Gene editing and designer babies experiments face global moratorium

Call for halt to human gene editing and designer babies experiments

Call for halt to human gene editing and designer babies experiments

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

Johns Hopkins researchers find genetic cause of olfactory neuroblastoma

Johns Hopkins researchers find genetic cause of olfactory neuroblastoma

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

UCL and AIIMS collaborates to increase academic and student exchange

UCL and AIIMS collaborates to increase academic and student exchange

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

Intense exercise, fasting and hormones can enhance waste-protein removal, study shows

Intense exercise, fasting and hormones can enhance waste-protein removal, study shows

Newly developed gene therapy helps decelerate aging process

Newly developed gene therapy helps decelerate aging process

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

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