The Spring 2018 issue of Directions is now available online. Check out our Support & Care publications page to download past issues of Directions and Cure SMA’s Care Series booklets. Our biannual newsletter builds our community by sharing updates, stories and experiences. Families from all over the world connect with each other through Cure SMA […]
Spring 2018 Issue of Directions Now Available Read More »
Cure SMA and Cytokinetics are very pleased to announce that Cytokinetics has committed to a National Platinum Partnership for 2018. Cytokinetics will be supporting several of Cure SMA’s awareness, education, and fundraising initiatives for spinal muscular atrophy (SMA), at both the local and national level. As part of their partnership, Cytokinetics is sponsoring the 2018 Annual
Cure SMA and Cytokinetics Announce National Platinum Partnership for 2018 Read More »
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 advanced by the efforts of Adrienne Vollmer, Graham’s mother, and State Representative Doug Gutwein, the bill’s
Indiana Becomes Fourth State to Adopt Newborn Screening for Spinal Muscular Atrophy Read More »
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 research and newborn screening. The visits also emphasized the
SMA Advocates Meet With House and Senate Offices Read More »
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 patient reported outcome measures for use in SMA clinical trials and clinics.
Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester Read More »
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 [the website] where we felt the most amazing support.
Uniting the SMA Community Through Local Programs and Support Read More »
Invitae Corporation recently announced that it will partner with Biogen to offer genetic testing at no charge to patients who may have spinal muscular atrophy (SMA). The program is called SMA Identified, and is intended to more quickly diagnose patients who may have SMA, or confirm a clinical diagnosis of SMA. The test also reports
Invitae Partners with Biogen to Offer Free Genetic Testing for SMA Read More »
Richard Finkel, M.D., the chief of neurology at Nemours Children’s Hospital in Orlando, Fla., was honored with the American Academy of Neurology’s 2018 Sidney Carter Award in Child Neurology, in recognition of his achievements in treating children with muscular dystrophy (MD) and spinal muscular atrophy (SMA). Dr. Finkel’s presentation in receipt of the Sidney Carter
The Phase 1, open-label, dose-escalation trial was designed to evaluate the safety and tolerability of AVXS-101 in patients with SMA Type 1. The key measures of efficacy were the time from birth to an event and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor
AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting Read More »
On Thursday, April 26, more than 70 chapter leaders, event organizers and board/committee members, representing 24 states and DC, will gather in Washington DC for two days of networking, learning, and advocacy. The activities include a day of advocacy on Capitol Hill, meeting with House and Senate offices, and a day of training and information
SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate Read More »
