June 2024

Spring 2024 Issue of Compass Now Available

The Spring 2024 issue of Compass is now available online.   This year, we are proud to announce six new research grants, totaling $750,000, to further our mission to drive breakthroughs in the treatment of spinal muscular atrophy (SMA). Basic research is the first step in developing new treatments and ultimately, a cure for spinal […]

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SMA Researchers and Clinicians from Around the World Gather at the 28th Annual SMA Research & Clinical Care Meeting

During the first week of June, SMA researchers and clinicians from around the world met in Austin, Texas, for the 28th Annual SMA Research & Clinical Care Meeting. Their common goals were to accelerate the pace of research into SMA treatments and achieve excellence in SMA clinical care. The annual meeting provides an opportunity for

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Check Out Cure SMA’s 2023 State of SMA Report

Cure SMA is pleased to announce the launch of the third State of SMA report. The annual data report highlights data from Cure SMA’s three databases: a patient-reported database with data from over 10,000 affected individuals worldwide that also incorporates longitudinal data from our annual Community Update Survey; the SMA Clinical Data Registry (CDR) that

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Cure SMA Announces Additional $750,000 in Basic Research Funding

At the Annual SMA Research and Clinical Care Meeting, Cure SMA highlighted six new basic research grants totalling $750,000. Cure SMA also announced another $750,000 will be awarded for additional research. The recently awarded projects will investigate new targets for drug discovery. With more than $85 million in funding to date in support of basic

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Five-Year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of SMA Achieved or Maintained the Ability to Sit, Stand or Walk

Summary of Data Presented After 5 years of treatment, 91% of children were alive — without treatment, children with Type 1 SMA would not be expected to live past 2 years of age. 96% of Evrysdi-treated children could swallow, 80% could feed without a feeding tube and 59% could sit without support for at least

Five-Year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of SMA Achieved or Maintained the Ability to Sit, Stand or Walk Read More »

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