News

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which included children treated with Zolgensma at older ages than those reported in clinical trials, suggested…

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…

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,…

As we’ve discussed in previous articles, most patients with SMA sit in a wheelchair all day, therefore increasing the likelihood of experiencing increased swelling in their feet and ankles. For able-bodied individuals, when they walk, their legs are in a gravity dependent position, and there is a natural…

[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…

[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…

[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…

[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…

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…

[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 look at what’s ahead for SMA,…