A new campaign is seeking to raise awareness of key motor development milestones for infants that, if missed, could mean spinal muscular atrophy (SMA) or other neuromuscular conditions. It’s been launched by Novartis Gene Therapies in collaboration with a diverse group of experts in the United Kingdom. Called…
Evrysdi (risdiplam), Roche’s oral therapy for spinal muscular atrophy (SMA), will be available to select patients in the U.K. before its potential regulatory approval through an Early Access to Medicines Scheme (EAMS). Eligible patients include those with type 1 and 2 disease, ages 2…
Two years after acquiring the gene therapy company AveXis, Novartis has renamed it Novartis Gene Therapies to underscore the potential value of developing such treatments for genetic diseases. This decision was based partly on the success of Zolgensma, originally developed by AveXis, as a…
I have never been a morning person. My family, friends, co-workers, and pretty much anyone that has ever met me can tell you this is true. But on rare occasions, I’ll wake up quickly without wishing for just a few more moments of sleep. Last week was one such occasion.
SMA Europe has launched an alliance that is demanding that, by 2025, newborn screening programs in Europe include a test for spinal muscular atrophy (SMA). The new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance) was launched as a final initiative to mark this…
[Editor’s Note: This is part of a series of articles about the discovery and development of Evrysdi — the newly approved disease-modifying therapy for spinal muscular atrophy and the first oral, at-home one — as well as the scope of SMA issues and treatments. Here, we focus on…
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi — SMA’s newly approved disease-modifying therapy and its first oral and at-home one — as well as the scope of SMA issues and treatments. Here, experts advise against comparing treatments head-to-head…
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi — SMA’s newly approved disease-modifying therapy and its first oral and at-home one — as well as the scope of SMA issues and treatments. Here, experts discuss what patients might expect…
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi — SMA’s newly approved disease-modifying therapy and its first oral and at-home one — as well as the scope of SMA issues and treatments. Here we again look at what might…