Scotland has become the first part of the UK to introduce screening for Spinal Muscular Atrophy (SMA) Type 1, which is a triumph for Jesy Nelson after she started campaigning for better testing of the ...

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 ...

Modulating the activity of a kinase in motor neurons may help mitigate mitochondrial defects and other symptoms of spinal muscular atrophy, offering a new therapeutic avenue for the devastating ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here are ...

PHILADELPHIA, Feb. 26, 2026 /PRNewswire/ -- Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage global genetic medicines company, announced today that the first patient has been dosed in the Phase ...

Jesy Nelson is opening up about her babies’ health. After the Little Mix alum’s mom noticed that her 8-month-old twin daughters Ocean and Story—whom she shares with fiancé Zion Foster—were showing ...

Learn how tracing the brain’s separate origins helped researchers grow nerve cells that could aid studies of ALS and spinal muscular atrophy.