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 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio Topline results from a phase 3 clinical ...

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 ...

Spinal muscular atrophy (SMA) is a severe neurological disease caused by reduced survival of motor neuron (SMN) protein levels. There is presently no cure for the condition, although current therapies ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

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 ...

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. Genetic factors are very important in ...

Charlie Zerzan refuses to let SMA define him, using wheelchair basketball to chase championships and inspire others.

The Chaparral Football Car Wash Fundraiser for SMA is happening Saturday, October 25, from 11 a.m. to 1 p.m. at Chaparral ...

When we think about genetic diseases, the ones that usually come to mind are cystic fibrosis, Fragile X and, of course, Tay-Sachs disease. One disease that many people are not aware of is spinal ...