The ultimate goal at FSMA is to accelerate the discovery of an effective treatment for SMA. To reach this end, we have institued a research-funding program with three distinct parts: basic research grants, drug discovery projects, and clinical trial initiatives.   We are also making a substantial investment in promising alternative therapies, such as stem cell therapy to broaden and diversify our chances for success.

Currently, FSMA funds basic research through our annual grant program, which accepts applications each September.  In drug discovery, we have two ongoing programs focusing on small molecule compounds that enhance SMN levels by complimentary mechanisms.  In addition, SMA has solely funded the Project Cure SMA Clincal Trials Network, which has completed 3 clinical trials to date and has 2 more ongoing. 

In addition, each year Families of SMA hosts and funds the largest SMA research conference, which brings together scientists from around the world to discuss the latest breakthrough in SMA.  This meeting is held in conjunction with the FSMA Family Conference, making the meeting is a wonderful opportunity to meet the patients and families who benefit from the research.