SRK-015 has received orphan drug status designation by the U.S. Food and Drug Administration (FDA) to treat muscle atrophy in patients with spinal muscular atrophy (SMA). SRK-105 is Scholar Rock’s lead product ... Read more
Editor’s note: Columnist Michael Casten continues his “Family Perspectives” series exploring how different people who surround Ella tackle the diagnosis of her SMA. Read an introduction to the series here. What is ... Read more
AVXS-101 has been awarded the SAKIGAKE designation in Japan for the treatment of spinal muscular atrophy (SMA) type 1. This designation will speed the therapy’s development and review process and allow ... Read more
According to scientists, new research on ALS may shed light on the mechanisms underlying spinal muscular atrophy.
Two RNA binding proteins, TDP-43 and hnRNP A1, are abnormal in certain cases ... Read more
Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and Pompe disease. Acsadi, head of ... Read more
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