Our Impact
FDA Approves Spinraza for SMA
On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA. […]
Read More ›Cure SMA Participates in a Special FDA Listening Session
Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order […]
Read More ›Cure SMA Announces Additional Conference Support for Newly Diagnosed Families
Thanks to the generous funding provided by the Erin Trainor Memorial Fund, Cure SMA is now offering additional support for newly diagnosed families to attend […]
Read More ›Help Us Shape the Future Together, and Reach our Goal of $750,000
This has been an historic year for the SMA community. On October 28, our pharmaceutical partners at Biogen and Ionis announced that their New Drug […]
Read More ›Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia
Spinal Muscular Atrophy: Disease Mechanisms and Therapy, the first comprehensive encyclopedia of SMA, has been published by Elsevier. Over thirty five of the book’s authors […]
Read More ›Cure SMA Launches Insurance Coverage and Payment Policy Project
Earlier this year, Cure SMA initiated an insurance coverage and payment policy project, in partnership with District Policy Group. The goal of this project is […]
Read More ›Cure SMA to Host Webinar on the New Drug Application Process
On Tuesday, December 6, Cure SMA will be hosting a one-hour webinar on the New Drug Application (NDA) process. The webinar will be held at […]
Read More ›6th Annual Congressional Dinner Furthers SMA Advocacy Work
This year’s 6th Annual “Hope on the Hill” Congressional Dinner will be held on Tuesday, November 29, bringing together families, government, and industry for an […]
Read More ›SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient […]
Read More ›Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
Twice this year, Cure SMA has had the opportunity to testify before the federal Advisory Committee on Heritable Disorders on the need for newborn screening […]
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