News

[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 talk…

Although both Evrysidi (risdiplam) and Spinraza (nusinersen) work to correct the same issue in spinal muscular atrophy (SMA), they do so through different mechanisms. SMA is caused by mutations in the SMN1 gene, preventing the production of…

[Editor’s Note: This is one in a series of articles about the discovery and development of Evrysdi, the newly approved disease-modifying therapy for spinal muscular atrophy (SMA), as well as the scope of SMA issues and treatments. Here, we spoke with leaders at PTC Therapeutics about the…

[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, at-home one, as well as the scope of SMA issues and treatments. Here, we talk with the family of a young…

From candlelighting events to storytelling, efforts to mark Spinal Muscular Atrophy Awareness Month, observed each August, are underway to heighten awareness of the rare genetic disease and raise funds to fight it. Supporters are participating in a range of activities such as changing their social media profile photo,…

[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 talk with the chief of the…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children…

[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 talk with scientists about how…

The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first oral and at-home treatment for adults, children, and infants 2 months of age and older with all types of spinal muscular atrophy (SMA). With this action, Evrysdi — marketed by…

A clinical trial testing oral amifampridine phosphate in people with spinal muscular atrophy (SMA) type 3 who are able to walk is fully enrolled, Catalyst Pharmaceuticals announced in a press release. Marketed under the brand name Firdapse, amifampridine phosphate is approved in the U.S. and the…