Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives 

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 Annual SMA Research & Clinical Care Meeting (June 5 – 7, 2024). 

Our poster presentations feature data from research initiatives launched by the Cure SMA Industry Collaboration (SMA-IC). The SMA-IC is a partnership that brings together pharmaceutical companies, Cure SMA, and other patient advocacy groups to share information, ideas, and data. The collaboration works to address scientific, clinical, and regulatory topics that are critical to advancing SMA drug development and will benefit the broader SMA community. The ongoing goals of the SMA-IC include: 

  • Update of the Cure SMA Toolkit on Best Practices for SMA Physical Therapists and Clinical Evaluators  
  • Creation of a Global Set of Patient Centered Outcomes for SMA  
  • Development of a Survey to Identify Unmet Needs and Access Disparities for SMA Treatment and Care  
  • Development of a Survey to Study the Diagnostic Journey of Adult Onset SMA  
  • Launch of the 8th Annual Cure SMA Community Update Survey 
  • Development of Pan European Survey on Treatment Decision Making 
  • Development of a Guide on Patient-relevant Design of SMA Clinical Trials  

Below is a re-cap of the posters presented this spring and additional posters to be presented at the Cure SMA Annual SMA Conference and Annual Research & Clinical Care Meeting in June. 

About the Cure SMA Industry Collaboration 

The Cure SMA Industry Collaboration (SMA-IC) was established in 2016 to leverage the experience, expertise, and resources of pharmaceutical and biotechnology companies, as well as other nonprofit organizations involved in the development of spinal muscular atrophy (SMA) therapeutics to more effectively address a range of scientific, clinical, and regulatory challenges. Current members include Cure SMA, Scholar Rock, Biogen, Biohaven Pharmaceuticals, Genentech/Roche, Novartis, Alcyone Therapeutics, NMD Pharma, and SMA Europe.  

Funding for this research was provided by the 2023 SMA-IC; members include Cure SMA, Biogen, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, Genentech/Roche, and SMA Europe.  


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