May 22, 2018
Biogen has released the following community statement on Spinraza access and new research data.
Dear Members of the SMA community,
Thank you for your continued support, efforts to raise awareness about SMA, and participation in clinical trials and studies as we seek to expand...
May 21, 2018
The Family Friendly Research Poster Session allows for one-on-one interactions between families and researchers. Cure SMA has invited researchers, who are attending SMA Researcher Meeting, to present family friendly research posters. During the event, families are encouraged to rotate to the...
May 17, 2018
Cure SMA and Cytokinetics are very pleased to announce that Cytokinetics has committed to a National Platinum Partnership for 2018....
May 15, 2018
Dear SMA Community,
Following the announcement last month, we wanted to let you know that AveXis (the gene therapy company developing a new approach to treat SMA known as AVXS‐101) is
May 14, 2018
On Friday, Governor Eric Holcomb held a signing ceremony for Indiana House Bill 1017, adopting newborn screening for spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID).
Dubbed “Graham’s Bill” in honor of Graham Vollmer, the bill was largely...
May 9, 2018
On Thursday, April 26, Cure SMA traveled with more than 70 spinal muscular atrophy advocates to Washington DC to meet with congressional offices. The advocates represented 24 states and DC. The purpose of the advocacy day was to advance life-changing opportunities for our community through SMA...
May 8, 2018
Cure SMA has awarded a $150,000 research grant to Chad Heatwole, MD, at the University of Rochester, for his project, " Development of a Clinically Relevant Outcome Measure for Pediatric SMA Therapeutic Trials.”
Dr. Heatwole and his team are working to develop SMA-specific...
May 7, 2018
AveXis has provided the following community statement on the SPR1NT trial.
Dear SMA Community,
AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT. SPR1NT is a...
May 2, 2018
After her son William's SMA type 2 diagnosis in April 2001, Heidi Johnson’s doctor immediately showed her the Cure SMA (then Families of SMA) website and said to look for support within the SMA community.
“After arriving home, we took the doc’s advice and opened up...