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…

[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, including…

[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 an Indiana man who began…

August has always been one of my favorite months. Not only is it an ideal time for enjoying lots of fresh air, but it also happens to be SMA Awareness Month. The entirety of August is dedicated to informing the world about this rare disease…

[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 with…

[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 expertise,…

There’s no need to dwell on the insanity that has defined 2020, so I’m skipping right on over to this month. I think it’s August. August is SMA Awareness Month, an opportunity to flood the universe with all things SMA for 31 days. Those in 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 boy…

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[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 SMA…