Amyotrophic lateral sclerosis (ALS), or Lou Gehrig disease, is a progressive, always-fatal neuromuscular disease characterized by motor neuron degeneration in the brain and spinal cord. As upper and ...

A 5-year-old British boy with the rare disease spinal muscular atrophy (SMA) has miraculously begun walking four years after receiving the world’s most expensive gene therapy. On the 31st, the BBC ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

Children with spinal muscular atrophy (SMA), a rare autosomal recessive disease that results in progressive muscle weakness and atrophy, can suffer the concerning consequences of dysphagia, which ...

Biogen said U.S. and European regulators will review its applications seeking approval of a higher-dose regimen of its Spinraza drug for the neuromuscular disease spinal muscular atrophy. The ...

Nicole Moore had contemplated getting her driver’s license before, but the progressive nature of spinal muscular atrophy, a neuromuscular disease she was diagnosed with at about 15 months old, held ...

Mutations that lead to muscle atrophy can be repaired with the gene editor CRISPR-Cas9. A team led by ECRC researcher Helena Escobar has now introduced the tool into human muscle stem cells for the ...

A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.