Cure SMA is pleased to announce that we are accepting grant applications for funding of research projects and postdoctoral fellowships, under a competitive review by our Scientific Advisory Board (SAB).  Please click here to view the SAB roster.

As we are now able to return to in-person activities for our SMA community, including conferences and fundraising events, we are very excited to be able to restart funding for this very important area of our mission.  Funding and leading the early research on understanding the disease and identifying treatment targets has led to the significant changes and outcomes that are now occurring for individuals and families impacted by SMA.

But our community has new needs and there is more work for us to do.  Our funding and commitment to early research will increase over the coming years to give us the answers we need.

The Importance of Continuing to Invest in Basic Research

  • In the past two decades, Cure SMA has invested $15 million in funding for 128 basic research grants that have furthered our understanding of SMA.
  • Cure SMA provided early funding for basic research projects that resulted in the development of our now approved genetically targeted drugs.
  • While the currently approved therapies dramatically alter disease, there remains significant unmet needs especially for our older and the symptomatic SMA population.
  • Continued basic research ensures a robust pipeline to now identify potential therapeutic avenues that will work in different ways and in combinations to address these needs and help restore strength and function.

What Are The Key Areas of Research We Want to Understand?

  • Non-SMN targets. We’re looking for other systems, pathways, and processes that can serve as the basis for an SMA treatment. We’re asking how we can best measure the effectiveness of these “non-SMN” approaches.
  • How to best utilize both SMN-enhancing approaches and non-SMN approaches. We’re asking how these different approaches can be used in combination to provide treatments for all ages and stages of SMA.
  • The role of SMN protein and when and where it is needed. We’re investigating what critical functions it performs, where it is lacking in the body tissues of those with SMA, and how quickly it needs to be replaced to provide benefit for those with SMA.

What is the Cure SMA Funding Model? 

The Cure SMA research-funding model is firmly based on the philosophy of expert and independent review and oversight of research projects. The Cure SMA strategy consists of having expert advisors review, select, and then oversee the research projects that we fund. This system has many advantages. Primarily, it ensures that Cure SMA funded the most promising research. In addition, funded projects are then run in a professional manner with the guidance of world-class experts. This type of system is the gold standard for effective scientific funding worldwide and is used extensively by both governments and nonprofit groups. It is often referred to as “peer review”, which means scientists working in similar areas judge each other’s work.

Never a Better Time to Invest in SMA Research!

Due to the generous support of Richard and Jane Nunemaker, gifts to these peer-reviewed research commitments will be matched up to $250,000!  Your investment in SMA research will focus on ways to enhance muscle strength and function.  Your investment may also have an impact on nerve muscle connections and regenerating nerves. And your gift will be matched up to $250,000.  Thank you to the Nunemaker’s for bringing this research closer to reality with their gift.  If you want your gift match, write “Research Match” on the check memo or on the donation form.  And thank you for your continued support.