Industry Updates
Biogen Shares DEVOTE and RESPOND Study Updates
Biogen recently shared that they have completed enrollment in the global DEVOTE and RESPOND clinical studies. Check out the full SMA Community Statement here.
Read More ›Genentech Shares 1-Year Primary Analysis from RAINBOWFISH
Last week, Genentech, a member of the Roche Group, presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and […]
Read More ›Novartis Gene Therapies SMA Community Update
Dear SMA Community, It was a pleasure to be able to join so many of you at the Cure SMA Annual Conference in Orlando this […]
Read More ›SMA Highlights and Updates from Biogen
Dear SMA Community, As we reflect on SMA Awareness Month, we take this opportunity to celebrate all those who are a part of the SMA […]
Read More ›NMD Pharma Initiates Phase II Trial for SMA
NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has […]
Read More ›Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
Today, Scholar Rock announced that enrollment in their Phase 3 SAPPHIRE trial is now complete. The Phase 3 trial will evaluate the safety and efficacy of […]
Read More ›Genentech Releases SMA Community Letter
Genentech recently issued an SMA community statement highlighting updates from the 2023 SMA Research & Clinical Care Meeting. These updates included: Investigating risdiplam treatment post […]
Read More ›Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration
Cure SMA is pleased to announce the launch of an expanded Phase 3 of the Real World Evidence Collaboration. The collaboration was established to leverage […]
Read More ›New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy
Biogen Inc. announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA […]
Read More ›Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk
Genentech, a member of the Roche Group, announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing […]
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