Front Page News
Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA
For the past several years, we have been working to bring your voices, experiences, challenges and hopes to inform regulatory authorities as key decisions are […]
Read More ›Cure SMA Announces Over $5 Million in New Research and Care Funding
Cure SMA is pleased to announce $5 million in new research funding over the next 12 months. This funding will be used strategically to help […]
Read More ›Registration is Now Open for the 2018 Annual SMA Conference!
Registration is now open for the 2018 Annual SMA Conference! This year’s conference will be held at the Hilton Anatole Hotel in Dallas, TX, from […]
Read More ›Cure SMA Meets in Utah with State and Federal Elected Officials to Support Newborn Screening
On Tuesday, Cure SMA staff members and family advocates, and clinicians from the University of Utah, met with Senator Orrin Hatch (R-UT) to discuss newborn […]
Read More ›Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)
Genentech/Roche has provided the below community statement with clinical trial updates for SUNFISH (Type 2/3). The first patient is now enrolled in SUNFISH, as the […]
Read More ›Recording of the September Webinar on Spinraza Updates Now Available
A recording of last week’s webinar updating the community on Spinraza access and administration sites is now available online. A PDF of the webinar presentation is also […]
Read More ›AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and […]
Read More ›Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy
To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , […]
Read More ›Newborn Screening Advocacy Update
At our Annual SMA Conference in July, we announced a grassroots campaign to implement newborn screening for SMA in all 50 states. We know that […]
Read More ›Novartis Releases Update on LMI070 (Branaplam) Clinical Trial
Novartis has released the following update for the LMI070 (branaplam) clinical trial, on behalf of the program team. Dear SMA community, For years, our researchers […]
Read More ›