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.
We are pleased to share the latest community update from Roche with several topics that are relevant to our SMA Community.
We are pleased to share the latest community update from Roche, announcing that the CHMP (Committee for Medicinal Products for Human Use) at the European Medicines Agency (EMA) has recommended the approval of an indication extension for risdiplam (Evrysdi), for all ages, from birth.
Biocad, a Russian biotechnology company, shares a community statement on its gene therapy product, ANB-004, which is being developed to treat SMA.