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.
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

New gene therapy for Pompe disease may replace currently available treatments

New gene therapy for Pompe disease may replace currently available treatments

Synthetic molecule appears to reverse Alzheimer's-related neurological damage

Synthetic molecule appears to reverse Alzheimer's-related neurological damage

TUM researchers decipher secret behind cohesion of heart muscles

TUM researchers decipher secret behind cohesion of heart muscles

MGH researchers identify gene mutations linked to rare congenital condition involving lack of nose

MGH researchers identify gene mutations linked to rare congenital condition involving lack of nose

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

UNC Catalyst aims to provide knowledge and research tools to tackle rare diseases

UNC Catalyst aims to provide knowledge and research tools to tackle rare diseases

Researchers find striking differences between countries, age groups in access to proper care for DMD

Researchers find striking differences between countries, age groups in access to proper care for DMD

Study reports volume and cost of in-home care for children with special medical conditions

Study reports volume and cost of in-home care for children with special medical conditions

Study tracks parents' unpaid time assisting children with special health care needs

Study tracks parents' unpaid time assisting children with special health care needs

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

MDA celebrates FDA approval of new spinal muscular atrophy drug

MDA celebrates FDA approval of new spinal muscular atrophy drug

TSRI scientists develop new methods to design precision medicines against disease-causing RNAs

TSRI scientists develop new methods to design precision medicines against disease-causing RNAs

Cedars-Sinai receives $7.3 million grant to test safety of novel cell-based therapy in treating PAH

Cedars-Sinai receives $7.3 million grant to test safety of novel cell-based therapy in treating PAH