"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...

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 ...

SMA is a genetic condition that impacts motor neurons, leading to muscle weakness that affects essential functions like breathing and movement. Treatments like Spinraza and Zolgensma can help manage ...

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 ...

Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...

Spinal muscular atrophy (SMA) Type 1 is a rare but serious genetic condition that weakens muscles and can make basic activities like eating and breathing hard for babies. Early treatment—especially ...

"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 ...

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.

PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...

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