A multidisciplinary team at Medcare delivered the therapy, led by its Gene Therapy Unit clinical lead, Dr. Vivek Mundada, ...

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 ...

Cenrifki prevents MS disease progression and Itvisma delivers a healthy gene-to-cell nuclei, addressing the root cause of SMA.

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

Spinal muscular atrophy (SMA) is a rare, genetic condition that, across all types, affects ~1,600 people in the UK.1 It can cause muscle weakness which gets worse over time, causing movement, ...

Ashley Webb, MD, Program Director, Pediatric Neurology, at the University of Texas Health Science Center at Houston McGovern Medical School, discusses the need to make genetic therapy more accessible ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

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 ...

'My life is complete.’ ...

Ellie Kerrigan, who has spinal muscular atrophy, shared her journey through Alabama sorority recruitment on TikTok ...

This marks a significant advancement in gene therapy, as this patient is the second adult SMA patient worldwide to be treated ...