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 »
Cure SMA is extremely pleased to announce a generous $620,000 gift has been made to the organization. The donation was made anonymously in honor of William N. Kanehann. Billy had SMA and died in 2013 at the age of 23. We are grateful for this amazing donation in memory of his life. This gift will
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Biogen has provided the following community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA. Dear Members of the SMA community, This past December marked one year since the FDA approval of SPINRAZA® (nusinersen), the first and only treatment option for children and adults living with SMA in the U.S. Since
Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research Read More »
Cure SMA is pleased to announce the second round of grants from $450,000 in funding, awarded to a total of 9 sites to help increase capacity at SMA treatment centers across the US. In January 2018, Cure SMA selected five additional treatment centers, totaling $200,000, to receive a grant to further increase the number of
Additional Funding for SMA Care Centers Announced Read More »
Cure SMA is pleased to announce a slate of one-day local symposiums, beginning late spring and continuing into fall 2018. The symposiums will be held in 16 locations across 15 states across the US. Through these local symposiums, the SMA community will continue to share experiences, advocate for one another, and learn about the latest
Cure SMA Announces Local Educational Symposiums in 16 US Locations Read More »
The winter 2018 issue of Compass is now available online. This issue covers Cure SMA’s community survey, and reviews how data is used to improve research, care and coverage in the SMA community. Cure SMA Community Update Survey Cure SMA has been collecting data through our database and various projects over the past several years.
Winter 2018 Compass Now Available Online Read More »
The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) today recommended that newborn screening for spinal muscular atrophy be implemented nationwide. This decision is an important step toward our goal of having every baby born in the United States screened for SMA. The recommendation now goes to Health and Human Services Secretary Alex
