News
May 8, 2018
Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester
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...
Topics: Our Impact, Research, Front Page News
May 7, 2018
AveXis Issues Community Statement on the SPR1NT Trial
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...
Topics: Clinical Trials, Front Page News
May 2, 2018
Uniting the SMA Community Through Local Programs and Support
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...
Topics: Support & Care, About Us, Front Page News
May 1, 2018
Invitae Partners with Biogen to Offer Free Genetic Testing for SMA
Invitae Corporation recently announced that it will partner with Biogen to offer genetic testing at no charge to patients who may have...
Topics: Support & Care, Research, Front Page News
April 30, 2018
Dr. Richard Finkel, M.D., Honored with the American Academy of Neurology's 2018 Sidney Carter Award in Child Neurology
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...
Topics: Research, Front Page News
April 25, 2018
AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting
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...
Topics: Clinical Trials, Front Page News
April 24, 2018
SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate
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...
Topics: Community & Awareness, Advocacy, Front Page News
April 24, 2018
The Discovery of Spinraza
SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein—called survival motor neuron protein or SMN protein—that is critical to the function of the nerves that control our...
Topics: Our Impact, Research, Front Page News
April 23, 2018
There is Still Time to Complete Cure SMA's 2018 Community Survey
Dear Members of the SMA Community,
For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate...
Topics: Our Impact, Research, Front Page News
April 23, 2018
Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting
Biogen recently presented new data on Spinraza for the treatment of spinal muscular atrophy (SMA) during the 70th American Academy of Neurology (AAN) Annual Meeting, currently taking place from April 21-27 in Los Angeles, California. Both platform and poster presentations highlighted the...
Topics: Clinical Trials, Research, Front Page News