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 ...

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 ...

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 ...

MIAMI - Celine Domalski was busy riding her unicorn at her Miami Beach condo as she readied to walk in a very important fashion show. "I'm doing a fashion show," Celine told Lisa Petrillo. "Are you ...

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 ...

Struggling to swallow, producing excess saliva and drooling can in some cases be a warning sign of a motor neuron disease, according to health experts. Motor neuron diseases (MNDs) are a cluster of ...

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.

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 ...