Latest News
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 available for download. An update […]
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 Drug Administration (FDA) has notified […]
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 , “Spinraza and Advanced Therapies: a […]
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 newborn screening and early treatment […]
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 have been working hard to […]
Cure SMA Announces $5.05 Million in Research Funding for FY18
Over the past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded over $2.5 million in new research funding. This funding will be used strategically to help […]