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 our priorities to the FDA and regulators, provide insight into daily […]
Last night, Governor Eric Holcomb of Indiana signed HB 1017, adding spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID) to the state’s newborn screening panel. The bill makes Indiana the fourth state in the country to adopt permanent SMA screening, following Missouri, Utah and Minnesota. The legislation was sponsored by Representative Doug Gutwein, and
Indiana is the Fourth State to Adopt Permanent SMA Screening Read More »
Cure SMA has awarded a $150,000 research grant to Allison Ebert, PhD, at the Medical College of Wisconsin, for her project, “Role of astrocyte-produced miR-146a in SMA pathology.” SMA is caused by mutations in the SMN1 gene resulting in low levels of SMN protein, which is expressed in every cell in the body. The goal
Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin Read More »
When a loved one is affected by spinal muscular atrophy, whether recently diagnosed or living with SMA for a while, you want to make a difference in their life and in the lives of all those affected by SMA. At Cure SMA, we believe in the power of the SMA community working together. That’s why
Awareness is Best Raised When We Work Together Read More »
We are excited to welcome 20 new members to our Medical Advisory Council (MAC). These clinicians represent eleven specialties, including specialties that were not previously represented on the MAC. Our new members are: Alison Ballard, RN, MSN, Pediatric Nurse Practitioner and Neuromuscular Care Coordinator at Children’s Hospital Colorado in Denver, CO William Bell, BS Pharm,
Cure SMA Welcomes New Medical Advisory Council Committee Members Read More »
The spinal muscular atrophy community is celebrating significant progress toward adding SMA to newborn screening panels in several states. Indiana Poised to Become the Fourth State to Adopt SMA Screening In Indiana, HB 1017 has been passed by both the House and Senate, and is currently awaiting Governor Eric Holcomb’s signature. This bill would institute
SMA Newborn Screening Moves Forward in Indiana, Ohio and Georgia Read More »
Rare Disease Day is today, February 28! And we’re calling on our entire spinal muscular atrophy community to get involved. Rare Disease Day is important to us because it is a time for individuals impacted by a rare disease—or those whose loved ones are impacted—to come together. It is a time for us to raise
#ShowYourRare and Raise SMA Awareness on World Rare Disease Day Read More »
A recording of last week’s webinar updating the community on treatment access and clinical trials is now available online. A PDF of the webinar presentation is also available for download. An update on the status of Spinraza dosing across the US included the following information: As of December 2017, nearly 2,000 US patients had been
Last Week’s Webinar on Treatment Access and Clinical Trials Now Available Read More »
The Weisman Family Foundations recently awarded $65,800 to Cure SMA, in order to further new scientific research, and to help provide access to treatments, clinical trials and care. Each year, a generous donation is made by the Weisman Family Foundations. Through contributions from their foundation and associated family foundations, the Weisman family has given over
Cure SMA Receives Generous Gift from the Weisman Family Foundations Read More »
Cytokinetics is currently conducting a Phase 2 clinical trial of CK-2127107 – now referred to by the generic name, reldesemtiv – which is designed to assess the investigational agent’s effect on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA. The trial includes two cohorts of varying doses of reldesemtiv. Enrollment
Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018 Read More »
