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 ...
A couple whose son died from a rare genetic condition are encouraging others to take part in screening which they say could ...
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 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 ...
The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...
The Independent on MSN
What is spinal muscular atrophy?
What is SMA? The rare genetic condition that Jesy Nelson’s twins have - Former Little Mix star revealed her twin daughters had been diagnosed with Spinal Muscular Atrophy (SMA) Type 1 ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...
In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...
Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...
Newborn babies in Hull will be able to be screened for more than 200 rare genetic conditions as part of a national study to ...
Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...
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