Search Results: zolgensma
Approaches to Drug Development
Make today a breakthrough. Due to a mutation in the survival motor neuron gene 1 (SMN1), individuals with spinal muscular atrophy (SMA) don’t produce survival […]
Read More ›SMA Drug Pipeline
Make today a breakthrough. The SMA Drug Pipeline is how Cure SMA evaluates the success of our spinal muscular atrophy (SMA) research program. It identifies […]
Read More ›Drug Discovery
Make today a breakthrough. Drug discovery takes what we’ve learned about the causes and biology of spinal muscular atrophy (SMA) and utilizes it for basic […]
Read More ›Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 […]
Read More ›Cure SMA Releases Updated Drug Pipeline
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 […]
Read More ›Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of […]
Read More ›Community Statement: Quarterly Update from Novartis Gene Therapies
Dear Members of the SMA Community, Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in […]
Read More ›Community Statement from Novartis Gene Therapies
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, […]
Read More ›Nusinersen Research Updates Presented by Biogen
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 […]
Read More ›Community Statement from Novartis Gene Therapies
Dear Cure SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use […]
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