SMA Newsroom

Clinical trialsMar 13, 2023

Biohaven's Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy

Biohaven announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, for the treatment of spinal muscular atrophy (SMA). 

Clinical trialsSep 20, 2022

Community update Scholar Rock: SAPPHIRE clinical trial sites in Europe

A community update has been shared by Scholar Rock regarding the sites for SAPPHIRE clinical trials in Europe. 

The SAPPHIRE study intends to enroll approximately 204 individuals with SMA across Europe and the USA. The plan is to have European trial sites in Belgium, France, Germany, Italy, Netherlands, Poland, Spain, and the United Kingdom. 

Scholar Rock has begun enrolling patients in Europe, with the first clinical trial sites up and running in Spain. The team is working on opening additional sites.

ResearchClinical trialsJul 13, 2022

Community update Biogen: ASCEND Study

The ASCEND study is a global clinical trial initiated by Biogen to evaluate if patients who have previously received treatment with risdiplam, an approved SMA drug also known as Evrysdi® (risdiplam), and may not have achieved optimal clinical outcomes (e.g., improvement or delay in disease progression), may benefit from receiving a higher dose of nusinersen, also known as Spinraza® (nusinersen).