Neuromuscular Disease News and Research

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Targeting RNA repeats

Targeting RNA repeats

Gene therapy treats myotubular myopathy in dogs

Gene therapy treats myotubular myopathy in dogs

New research offers more insights into development of Kennedy's disease

New research offers more insights into development of Kennedy's disease

Researchers develop molecule that binds to GAA enzymes key to progress of Pompe disease

Researchers develop molecule that binds to GAA enzymes key to progress of Pompe disease

Combination of sugar and polyphenols may prevent occurrence of neurodegenerative disease

Combination of sugar and polyphenols may prevent occurrence of neurodegenerative disease

Study explores two new antibodies believed to cause myasthenia gravis

Study explores two new antibodies believed to cause myasthenia gravis

ResMed announces definitive agreement to acquire Brightree

ResMed announces definitive agreement to acquire Brightree

Identifying and avoiding wasteful or unnecessary medical tests

Identifying and avoiding wasteful or unnecessary medical tests

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

U of T study provides hope for treating ALS and FTD

U of T study provides hope for treating ALS and FTD

Researchers unlock structural details of key protein to treat neuromuscular disease

Researchers unlock structural details of key protein to treat neuromuscular disease

Novel wound closure technique may reduce complication rates for patients with scoliosis

Novel wound closure technique may reduce complication rates for patients with scoliosis

Chronic inflammatory demyelinating polyneuropathy commonly misdiagnosed

Chronic inflammatory demyelinating polyneuropathy commonly misdiagnosed

Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

Isis Pharmaceuticals, AstraZeneca to develop antisense therapies for cardiovascular, metabolic, renal diseases

NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen

NEJM publishes positive clinical results from Phase 2 clinical study of volanesorsen

Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Akcea Therapeutics obtains FDA Orphan Drug Designation for volanesorsen

Akcea Therapeutics obtains FDA Orphan Drug Designation for volanesorsen

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Immune system linked to death of motor neurons in Amyotrophic lateral sclerosis

Immune system linked to death of motor neurons in Amyotrophic lateral sclerosis