Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by: | • | Funding and advancing a comprehensive research program | | • | Supporting SMA families through networking, information and services | | • | Improving care for all SMA patients | | • | Educating health professionals and the public about SMA | | • | Enlisting government support for SMA | | • | Embracing all touched by SMA in a caring community |
Our vision is a world where Spinal Muscular Atrophy is treatable and curable.
In
2009, the entire SMA community – families, patients, researchers,
clinicians – will celebrate the 25th Anniversary of Families of SMA.
The story behind this anniversary – of its progress and accomplishments
– is a story about dedication and determination. A small group of
parents started Families of SMA in 1984. They wanted to raise funds
for SMA research to cure the disease, and support all affected families. Back
then, very little was known about Spinal Muscular Atrophy. It was
frequently misdiagnosed. Very little research was being conducted. No
one knew the cause of the disease let alone how to find a treatment and
a cure. There were no family support services and no clinical trials.
Patients and families affected by SMA were on their own and had little
hope. Today, FSMA has a different story to tell: Families now
have hope. We have raised over $50 million for SMA
research. Families of SMA funds and directs the leading SMA research
programs. Families of SMA has created hope for our community that did
not exist in 1984. Our support comes from generous individual
donations and numerous fundraising events held by volunteer families
and our chapters. Our successful
results and progress from basic research to drug discovery programs to
clinical trials provides real hope for families and patients: | • | Families of SMA has funded 5 multi-center clinical trials for existing drugs that have potential for SMA | | • | FSMA has directed and funded the leading new drug development program for a therapy specially designed to treat SMA. | | • | Families of SMA is building a pipeline of drug discovery programs based on our investments in basic research | | • | FSMA has invested significant resources into alternative approaches that show promise to cure SMA |
In 2009: | • | Over 900 Attendees at the Annual SMA Family and Professionals Conference | | • | Families of SMA research funding lead to the publication of 18 scientific journal articles in 2009 | | • | Families of SMA’s Quinazoline495 drug candidate for SMA received Orphan
Drug Designation from the FDA, and completed a pre-IND meeting | | • | Families of SMA’s Motor Neuron Replacement Program for SMA received
Orphan Drug Designation from the FDA, and completed a pre-IND meeting. |
We are a non-profit, 501(c)3 tax exempt organization (Federal ID# 36-3320440). Funds are
specifically directed to scientific, educational or literary purposes
in keeping with a charitable organization. Today we have 27 Chapters
throughout the United States and over 65,000 members and supporters.
We are a collaborative organization where families and friends and
researchers are all working together towards the same goal.
FSMA sponsors the largest Annual International Research Conference
devoted entirely to SMA research. Dedicated researchers gather from
around the world to share results and exchange ideas, fostering a
spirit of cooperation and moving us toward our goal of finding a
treatment and cure sooner.
At the same time as the research conference FSMA sponsors an Annual
Conference for Families to learn about the status of SMA research, gain
an understanding of the disease, share the latest in disease management
techniques, and network with other families and medical professionals. We
encourage everyone touched by SMA to join our organization. All
parents of children with SMA, all people directly affected with SMA,
all relatives, friends and colleagues of those affected, are welcome to
join us as we work together to find a treatment and cure for SMA. |
