We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 programs in clinical trials. An ever-increasing breadth of ...
We are pleased to share an update on Genentech's, a member of the Roche Group, initiation of MANATEE, a new global Phase 2/3 clinical study that aims to evaluate the safety and efficacy ...
Novartis announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold ...
Data from Genentech, a member of the Roche Group, recently announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety ...
Novartis recently announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). New late-breaker data from the completed two-copy cohort ...
Dear SMA Community, Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in the diagnosis, treatment, and care of those affected by SMA. We ...
Genentech, a member of the Roche Group, has shared new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with ...
Biogen has shared new data from the Spinraza (nusinersen) clinical development program aimed at optimizing outcomes for people with SMA and advancing understanding of the disease. Exploring Opportunities to Optimize Treatment in SMA ...
Scholar Rock provided the below community statement on data for apitegromab (SRK-015) following the distribution of this company press release. Dear Members of the SMA Community, We are pleased to share 12-month topline ...
Biogen recently presented data about ongoing studies of SPINRAZA (nusinersen) in patients with Spinal Muscular Atrophy. Updates included the NURTURE, ENDEAR, SHINE, DEVOTE, and RESPOND clinical trials. Oral presentation details: Title: Predictive Factors of ...
Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in SMA Scholar Rock recently shared 6-month interim analysis data from the TOPAZ Phase 2 clinical trial evaluating the potential of apitegromab, a highly ...
New 2-year data show Evrysdi continues to demonstrate improvement or maintenance of motor function in people aged 2-25 years with Type 2 or 3 SMA Genentech, a member of the Roche Group, recently ...
