Search Results: Zolgensma
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 […]
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 […]
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, […]
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 […]
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 […]
Biogen SMA Q1 2021 Community Statement
Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to […]
Cure SMA Offer Perspectives on Combination Therapy for SMA
New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible for people with SMA, allowing them […]
Cure SMA’s New Year Letter for 2021
Dear SMA Community, While 2020 did not pan out as we had expected, Cure SMA remains extremely optimistic about the future for our community in […]
Cure SMA Funds Over $9 Million in New and Ongoing Research in FY20
During our past fiscal year—from July 1, 2019 to June 30, 2020—Cure SMA funded more than $9 million in new and ongoing research and care […]
Cure SMA Immunizations Recommendations During Flu Season and Amidst the COVID-19 Pandemic
Contributors: Dr. Mary Schroth and Dr. Robert Graham Influenza (or flu) is a serious respiratory infection, and people of any age can get it. Since […]