The SMA community now has 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline. The FSMA research approach funds programs at early stages, and then partners with companies to take them through clinical trials. Supporting multiple programs gives different approaches for a SMA therapy, which increases the chances of success and accelerates the timeline to a treatment and cure. The key to developing a treatment and cure is to build upon this progress and add many more programs to the drug pipeline. Families of SMA has invested over $18 million in 6 drug programs to date.
Families of SMA also provides support for families and patients today, including important patient care information, critical medical equipment, and The Annual SMA Conference. The Annual SMA Conference is the largest gathering in the world of families affected by SMA and leading researchers and medical professionals who all come together to share their ideas and optimism for the future.
Advancing Research – The SMA Drug Pipeline:
-We now have 13 new SMA drug programs in development, including 3 in clinical trials.
-This pipeline has expanded from just 4 programs 5 years ago.
-Families of SMA has funded half of all the ongoing drug programs for SMA.
Supporting Families – The FSMA Equipment Pool:
-In the last year Families of SMA provided direct services to over 3,600 patients and families.
-We supply critical resources specially designed for SMA patients to live a healthy and comfortable life.
-Recently added medical equipment ranges from adaptive bathing systems to positioning strollers.
Giving Hope – The Annual SMA Conference:
-1,500 attendees, 3 times as many families and patients as 5 years ago.
-New specialized programs for newly diagnosed families and for professional care providers.
-Participation has grown to 14 companies, including 3 major pharmaceutical partners.
FSMA research progress provides us all with hope that one day we will live in a world without SMA.