Neuromuscular Disease News and Research

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Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

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

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

Creative solutions to enhance patient's point of view in neuromuscular research

Creative solutions to enhance patient's point of view in neuromuscular research

Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

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

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

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Researchers discover how nerve cells control movement

Researchers discover how nerve cells control movement

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Researchers discover new approach for treatment of myotonic dystrophy

Researchers discover new approach for treatment of myotonic dystrophy

KFU medical departments seek to find treatments for hereditary syndromes

KFU medical departments seek to find treatments for hereditary syndromes

Beaumont and GVSU team up to develop lifesaving cough-assist device

Beaumont and GVSU team up to develop lifesaving cough-assist device

Study identifies potential genetic link in sudden infant death syndrome

Study identifies potential genetic link in sudden infant death syndrome

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Researchers describe human blood-nerve barrier transcriptome for the first time

Researchers describe human blood-nerve barrier transcriptome for the first time

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients