Over the past few months, Cure SMA has announced a total of just over $2 million in new research funding. Our model is designed to fund both a breadth and a depth of projects, in order to continuing building up both a breadth and depth of programs in the drug pipeline. This includes funding for:
- Basic research, to investigate the biology and cause of SMA, in order to identify the most effective strategies for drug discovery; and to develop tools that facilitate SMA research.
- Drug discovery, to convert basic research ideas into practical new drug candidates.
- Clinical and regulatory research, to facilitate clinical trials for all types and stages of SMA, and to guide safe and effective drug candidates toward regulatory approval
- Clinical care, to understanding issues like breathing and nutrition that impact current quality of life for those affected by SMA.
- 20th Annual SMA Researcher Meeting, to bring together the leading SMA scientists, clinicians, and drug developer together to develop SMA research strategies.
If you missed any of our funding announcements, catch up on them in our news section using the links below. Each announcement includes a profile of the researcher who is receiving the grant, and explains more about their projects and how they might benefit the SMA community.
Basic Research
- $140,000 Grant to Christine Beattie, PhD, Ohio State University
- $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- $90,000 Grant to Antoine Cléry, PhD, ETH Zurich
- $30,000 Grant to Jocelyn Cote, PhD, University of Ottawa
- $70,000 Grant to Christine DiDonato, PhD, Lurie Children’s Hospital of Chicago
- $140,000 Grant to Megerditch Kiledjian, PhD, Rutgers
- $140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute
- $140,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
Drug Discovery
- $304,000 Grant to Livio Pellizzoni, PhD, at Columbia University
- $300,000 Grant to Imago Pharmaceuticals/Charlotte Sumner, MD
*Two additional drug discovery grants totaling $100,000 will be announced later this month.
Clinical and Regulatory Research
- $100,000 to help fund the launch of a collaborative industry consortium working on clinical trials, regulatory issues, and education
Clinical Care
- $50,000 Grant to Melissa Alderfer, PhD, Alfred I. DuPont Hospital for Children
- $50,000 Grant to Tariq Rahman, PhD, Alfred I. DuPont Hospital for Children
SMA Researcher Meeting
- $250,000 to bring the leading SMA researchers together from around the world
We thank the following families and foundations for their generous contributions toward these grants: The Jacob Isaac Rappoport Foundation toward Dr. Burghes, the Bugenske Family and Leo’s Pride toward Imago Pharmaceuticals/Dr. Sumner, and The Miller McNeil Woodruff Foundation toward Drs. Sumner and Alderfer. The grants to Drs. Cote and Kothary were funded by Families of SMA Canada.