31 Days of SMA: Laura Blair
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Ohio Study Finds Zolgensma Safe, Effective, Particularly in Younger Infants
Novartis’ gene therapy Zolgensma is generally safe and effective in promoting motor improvements in children with spinal muscular atrophy (SMA) treated up to age 2, according to a multicenter study. However, “if the children are older and heavier, there is a potential for more liver injury [a known side effect…
FDA Approves Catalent Facility to Aid in Zolgensma’s Production
The U.S. Food and Drug Administration (FDA) has given Catalent Biologics approval to aid in manufacturing products for use with AveXis’ spinal muscular atrophy (SMA) gene therapy, Zolgensma, at its Maryland facility. The FDA inspected the company’s commercial-scale gene therapy manufacturing center in June. Catalent and AveXis,…
FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA
The U.S. Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to SRK-015, an investigational treatment for spinal muscular atrophy (SMA), its developer, Scholar Rock, announced. This designation is given to therapies that are intended to treat serious or life-threatening disorders that…
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
SMA Registries Offer ‘Real-world Experience’ That Makes Best Treatment Possible
[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…
Comparing SMA Therapies Neither Easy Nor Fair, Clinicians Say
[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…
Treatment Is Key for All With SMA, But Gains Will Differ
[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…
Ways of Capturing Real-life Gains, True Value of SMA Therapy, Still Needed
[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…
Evrysdi for SMA Under Accelerated Review in Europe
The European Medicines Agency (EMA) has accepted and is now reviewing Roche’s marketing authorization application (MAA) requesting approval of Evrysdi (risdiplam) for treating spinal muscular atrophy (SMA). Given that Evrysdi previously received the EMA’s priority medicines designation for SMA, enabling accelerated assessment of its application, a decision may be expected…
