Spinal muscular atrophy (SMA) type 1 is a genetic condition in which the nerves that control movement, breathing, and swallowing become progressively weaker and smaller. SMA has five types with ...

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 ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

– Infants treated with Evrysdi maintained or continued to improve in measures of motor function, including their ability to sit without support for 5 and 30 seconds – – Evrysdi has proven efficacy in ...

According to researchers, the most prevalent risk factors in children with type 1 spinal muscular atrophy (SMA) are hypercalciuria and elevated urine specific gravity. Children with spinal muscular ...

Biomarkers for SMA were identified using LC-MS and machine learning, revealing differences in protein expression across SMA types. CNTN1 and NRXN3 proteins showed potential as prognostic biomarkers, ...

Spinal muscular atrophy (SMA) life expectancy varies between types. The most severe types of SMA have a life expectancy of less than 2 years, while less severe types do not impact life expectancy. The ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...

(RTTNews) - Novartis (NVS) announced new data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), an essential one-time treatment for spinal muscular atrophy or SMA.

A child was treated for a rare, potentially deadly genetic disorder while still in the womb — and now, she has survived past ...