Search Results: spinraza
New Clinical Trial Data Published in New England Journal of Medicine
Two new papers of clinical trial data for spinal muscular atrophy were published today in the November issue of the New England Journal of Medicine […]
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 , […]
SMA Documentary Premieres at 2017 Rhode Island International Film Festival
Life & Atrophy, a film about SMA, premiered at the Rhode Island International Film Festival on Saturday, August 12th. It was directed, produced, edited and […]
Centers for Medicare and Medicaid Services (CMS) Survey
On August 3rd, as part of multiple days of advocacy visits, Cure SMA staff met with leaders from the Medicare division of the Centers for […]
SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts
On August 2nd, members of the spinal muscular atrophy community kicked off SMA Awareness Month by meeting with a dozen Senate and House congressional offices […]
2017 SMA Researcher Meeting Summary: SMA Therapy Development
The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]
Summer 2017 Update to SMA Drug Pipeline is Released
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 19 active programs, including one approved therapy. 14 pharmaceutical partners. 5 […]
Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal
The journal Neuromuscular Disorders has published a paper reporting on the findings of an international workgroup convened to update the Standard of Care (SOC) for […]
The 2017 Annual SMA Conference is Here!
The 2017 Annual SMA Conference, now in its 29th year, is set to kick off on June 29 and continue into a weekend full of […]
Cure SMA Awards $140,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires, Argentina
Cure SMA and FAME (Families of SMA, Argentina) have awarded a $140,000 research grant to Alberto Kornblihtt, PhD, at the Universidad de Buenos Aires, Argentina, […]