Author name: Sarah McCall

SMA Highlights: Updates from Biogen

On July 12, 2024, Biogen shared highlights and updates from the 2024 Annual SMA Conference, including their collaboration with Delta Flight Products and their sponsorship of the Self-Expression Art Exhibit focused on travel. Read the full community statement here. “Cure SMA hosted yet another wonderful Annual SMA Conference where community members traveled from across the […]

SMA Highlights: Updates from Biogen Read More »

Clinical Research and Drug Development Session Highlights from 2024 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

Clinical Research and Drug Development Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting Read More »

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

Spring 2024 Issue of Compass Now Available Read More »

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

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

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

Check Out Cure SMA’s 2023 State of SMA Report Read More »

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

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

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 »

Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Diagnosis Considerations

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

Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Diagnosis Considerations Read More »

Novartis Gene Therapies Releases Spring SMA Community Letter

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,

Novartis Gene Therapies Releases Spring SMA Community Letter Read More »

Scroll to Top