Spinal muscular atrophy (SMA) is a leading cause of infant mortality. Most cases of the disease are caused by mutations in a gene called SMN1 that reduce the production of functional SMN protein, ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...

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 ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights recent advancements and ongoing clinical trials in the spinal ...

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by mutations in the SMN1 gene that reduce survival motor neuron (SMN) protein expression. Despite progress in the field that ...

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no ...

As their 8-month-old daughter prepares for gene therapy treatment, a Coon Rapids family is bracing for three months of ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights the current FDA-approved therapies for spinal muscular atrophy.

Spinal muscular atrophy affects all the body's muscles. For a long time, it was considered a disease caused solely by the ...

Novartis' Zolgensma is approved in over 51 countries, and more than 3,700 patients have been treated globally across clinical trials. Most patients in the SMART study maintained motor milestones ...

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals, Inc. (1228.HK), a China and U.S.-based global biopharmaceutical company committed to the research, development and ...

THE WOODLANDS, Texas, May 11 /PRNewswire-FirstCall/ -- Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that it was awarded a grant from the United States ...