Cure SMA is a non-profit organization dedicated to the treatment and cure of spinal muscular atrophy (SMA). They fund ground breaking research and provide families the support they need for today.
Working tirelessly in the present
Since 1984, Cure SMA has directed and invested in the research that has shaped the scientific community’s understanding of SMA and made today’s breakthroughs possible. With deep connections and expertise in both the patient and research communities, Cure SMA is uniquely positioned to direct funds to where they can make the greatest difference as quickly as possible.
The organization’s board and staff members possess deep expertise in every aspect of SMA, from the day-to-day realities of life with SMA to the nuances of care options. While working for a cure, the organization also works to support the children and families currently affected by the disease. In the last year, Cure SMA provided direct services to more than 3,600 families.
Racing toward the future
Cure SMA has invested more than $57 million in research and has funded two-thirds of all the ongoing new drug programs for SMA. In the last year alone, the organization funded 30 research programs, which represent a diversity of therapeutic approaches.
Through their comprehensive research model, Cure SMA supported the first drug program for SMA from initial research all the way to clinical trials. Cure SMA now has a dozen companies involved in SMA research, including some of the biggest pharmaceutical companies in the world.
Making real progress
There’s great reason for hope. The scientific world now knows what causes SMA and what needs to be done to develop effective therapies. The SMA community is on the verge of major breakthroughs that will strengthen our children’s bodies, extend life, and eventually lead to a cure.
Ten years ago, the organization had just one potential drug in the beginning stages of preclinical discovery. Five years ago, they had four. Today they have more than a dozen, including five now in clinical trials. This means the scientific community is getting closer and closer to an FDA-approved therapy for people affected by SMA.
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