SMA Newsroom

Back to Index
Clinical trialsSep 20, 2023

Community update from Scholar Rock

We are happy to share with you the latest update shared with us by Scholar Rock.

The company announced yesterday that it has completed enrollment of the SAPPHIRE Phase 3 trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam).

You can read full press release below.

ResearchTreatmentsClinical trialsAug 30, 2023

Community update from Roche

We are pleased to share the latest community update from Roche with several topics that are relevant to our SMA Community.

Clinical trialsJun 30, 2023

Scholar Rock announces new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with apitegromab.

Scholar Rock announces new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with apitegromab. 

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).