Higher blood levels of phosphorylated neurofilament heavy chain (pNF-H) — a marker of nerve cell damage — before beginning Spinraza (nusinersen) treatment are associated with greater motor improvements in children with spinal muscular atrophy (SMA) types 2 or 3. Notably, this association increased over time, reaching statistical significance at about…
Prenatal enrollment of newborns for spinal muscular atrophy (SMA) screening leads to faster results than recruitment after birth, according to findings from an Early Check pilot study in North Carolina. While most results were obtained within a timeframe that would allow treatment to begin before symptom onset — key to…
Abnormal increases in liver enzymes levels — suggestive of liver damage — and drops in platelet counts are common among spinal muscular atrophy (SMA) patients over 8 months old following treatment with Zolgensma, according to a small real-world study in Germany. These side effects, which can be severe in some…
Evrysdi (risdiplam) continues to improve survival and to allow infants with spinal muscular atrophy (SMA) type 1 to achieve key motor milestones, according to two-year data from the second part of the FIREFISH trial. “It is encouraging to see that infants continued to improve after 12 months of treatment, with twice as many…
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), safely and effectively improved or stabilized motor function in children and young adults with types 2 and 3 disease over one year, top-line data from the Phase 2 TOPAZ trial show. Notably, patients’ motor gains were either maintained or increased at…
The European Commission has approved Evrysdi (risdiplam) as the first oral and at-home treatment for adults, children, and infants 2 months and older with nearly all types of spinal muscular atrophy (SMA). Eligible patients include those with a clinical diagnosis of SMA type 1, 2, or 3 or carrying one to four…
The benefits of newborn screening (NBS) for spinal muscular atrophy (SMA) outweigh its disadvantages and challenges, according to a survey of parents and healthcare providers of screen-positive newborns in Australia. Notably, both groups supported the implementation of a newborn screening program, as well as its equal access across the…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Zolgensma safely and effectively halts disease progression in pre-symptomatic spinal muscular atrophy (SMA) infants, according to updated…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Zolgensma continues to safely and effectively prevent motor function decline for at least five years, allowing the achievement…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Evrysdi (risdiplam) continues to improve or stabilize motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3,…
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