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 […]
Spring 2024 Issue of Compass Now Available Read More »
Thank you to everyone who attended the 2024 Annual SMA Conference at the JW Marriott in Austin, Texas! It was an impactful weekend of opportunities to connect, learn, and have fun. We are happy to announce that there were over 2,260 attendees in total – our second-largest conference to date! It was an honor to
Thank You for an Amazing 2024 Annual SMA Conference! Read More »
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
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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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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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
Cure SMA is pleased to announce the publication of the first in a series of new resources to support the care of individuals living with Spinal Muscular Atrophy (SMA). As part of our ongoing efforts to drive care improvements for the entire SMA community, Cure SMA recently published, Spinal Muscular Atrophy (SMA) Update in Best
Novartis Gene Therapies recently released an update to the SMA community related to clinical trials and long term follow up studies of Zolgensma, in addition to recent publications. Read the full community statement here. “It has been wonderful to have seen so many of you at Summits of Strength and the Walk-n-Rolls already this year,
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Table of Contents What is SMA How Common is SMA Types of SMA Causes of SMA Carrier Testing Symptoms of SMA Diagnosis SMA Newborn Screening Treatment for SMA Outlook / Prognosis Living with SMA Latest Updates on SMA SMA Research SMA Resources and Programs Clinical Trials and Studies Common SMA Questions Next Steps for SMA
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Cure SMA is pleased to announce the participation of scientific leadership in the 2024 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference (March 3 – 6, 2024) and the 2024 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference (May 5 – 8, 2024). We will also present these research findings at the upcoming
