Higher levels of the GFAP protein in the cerebrospinal fluid are associated with better clinical response to Spinraza in SMA, a study found.

Amy Moffatt's four-year-old son, Oakley, has the most serious form of Spinal Muscular Atrophy (SMA), which causes progressive ...

Nelson has previously revealed that their twins, Ocean Jade and Story Monroe Nelson-Foster, have a rare genetic condition.

After confirming their life-altering diagnosis, the former Little Mix star has now shared a rare snap of her beautiful twins ...

We existed in our own world that day, absorbing information we never wanted to learn. At one point, while I was crying ...

SMA patients in Kerala urge government to allocate funds for free drug distribution to combat the rare genetic disorder.

During the first day of the 44 th Annual JP Morgan Healthcare Conference in San Francisco, a clear narrative emerged. With ...

NEW YORK – The European Commission has approved a high-dose regimen of Biogen's Spinraza (nusinersen), an antisense oligonucleotide for 5q spinal muscular atrophy (SMA).

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both ...

Sam agreed, and said: "Unless you've lived it, it's hard to understand it, so having a sibling who can not only understand, ...

Welsh mum Samantha Williams is "100%" backing a campaign to introduce NHS heel prick tests for SMA. Her son Lucian Neale, now ...