Infants with spinal muscular atrophy (SMA) type 1 receiving the therapeutic dose of Evrysdi (risdiplam) continue to improve and achieve motor milestones, according to two-year data from the first part of the Phase 2/3 FIREFISH clinical trial. Updated data from FIREFISH (NCT02913482) showed that nearly all…
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…
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…
My dad and I are night owls. We rarely leave the house before noon — and when we do, we complain the entire time, like a pair of sulky babies. My mom laughs at us, but it’s just how our brains are wired. We can fight it, and force…
[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…