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.
Weekly doses of steroids help hasten recovery in muscle injuries

Weekly doses of steroids help hasten recovery in muscle injuries

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

EPFL researchers discover protein that kick-starts gene expression in nascent embryos

EPFL researchers discover protein that kick-starts gene expression in nascent embryos

Actress Kiruna Stamell argues about gene editing with Dr Christopher Gyngell

Actress Kiruna Stamell argues about gene editing with Dr Christopher Gyngell

NIH scientists discover mitochondrial 'circuit breaker' that prevents damage to the heart muscle

NIH scientists discover mitochondrial 'circuit breaker' that prevents damage to the heart muscle

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

BMJ Innovations releases first collaborative definition of patient centricity co-created by AstraZeneca

BMJ Innovations releases first collaborative definition of patient centricity co-created by AstraZeneca

Researchers discover small protein essential for formation of skeletal muscle

Researchers discover small protein essential for formation of skeletal muscle

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Scientists develop novel technique to screen non-coding DNA of genome for diseases

Scientists develop novel technique to screen non-coding DNA of genome for diseases

New research reveals surprising culprit involved in self-destruction of axons

New research reveals surprising culprit involved in self-destruction of axons

SLU researchers aim to tackle diabetes and obesity by capturing benefits of exercise in pill form

SLU researchers aim to tackle diabetes and obesity by capturing benefits of exercise in pill form

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

New test could accelerate development of better treatments for Motor Neuron Disease

New test could accelerate development of better treatments for Motor Neuron Disease

Targeting RNA repeats

Targeting RNA repeats

New method that detects NMD efficiency could help in treatment of many diseases

New method that detects NMD efficiency could help in treatment of many diseases

Emory researchers reveal 'matchmaker' role for protein linked to SMA

Emory researchers reveal 'matchmaker' role for protein linked to SMA

Research suggests new type of congenital muscular dystrophy

Research suggests new type of congenital muscular dystrophy

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

Study discovers new form of congenital muscular dystrophy caused by faulty INPP5K gene

Study discovers new form of congenital muscular dystrophy caused by faulty INPP5K gene