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.

Click here to see the FSMA Results and Progress through 2010.

Click here for some quick facts about SMA.
Click here for information on the patient services and support we provide.
Click here for details on the research programs we fund.

About FSMA
In 1984, a small group of parents across the country banded together to support, comfort and educate each other about the devastating disease affecting their children; Spinal Muscular Atrophy.  Their purpose was twofold—to support families affected by SMA and to fund research leading to a treatment and eventually, a cure.   Families of SMA has grown from this courageous group of families to an international network of fundraising and support including 31 volunteer Chapters throughout the United States. 

Since 1984, our legacy is one of unparalleled research progress and patient support, from grassroots fundraising to global scientific leadership. Families of SMA has funded over $50 million of SMA research in three areas:  Basic Research, Drug Development and Clinical Trials.

Basic Research projects identify the most effective new strategies for SMA drug discovery and fuel the therapeutic pipeline.  This long-term investment ensures that every possibility can be explored in the quest for an effective SMA therapy.   Families of SMA has funded more than 140 basic research grants to 70 institutions around the world, resulting in many critical SMA breakthroughs including:
- Mapping and cloning of the SMA gene, SMN1;
- Identification of the SMN protein and its roles in the cell;
- Discovery of the back-up gene SMN2.

Traditionally it has been difficult to attract pharmaceutical companies to conduct research for orphan diseases like SMA, which have small patient populations with small potential for profit.  Therefore, Families of SMA has taken the strategy of providing seed funding to encourage biotech and pharmaceutical partners to engage in SMA drug research. Families of SMA has invested in five new Drug Development programs for therapies specially designed to treat SMA including: 
- Quinazolines to boost SMN2 expression from the back up gene;
- Tetracyclines to correct SMN2 splicing in the back up gene;
- Motor neuron replacement or cellular therapy;
- Gene Therapy to replace the lost SMN1 gene.

Families of SMA believes that it is critical to invest in a Clinical Trials infrastructure to reach our ultimate goal of finding a safe and effective therapy for SMA.  To this end, our investment in this area allows us to accomplish several critical steps on the path to our final goal:
- Establish validated protocols for clinical assessment in every SMA type;
- Implement clinical trial sites across the United States to facilitate novel drug trials, conducted for FDA approval.

Families of SMA has funded five multi-center clinical trials for existing drugs that have potential for SMA.

Along with funding and directing leading SMA research, Families of SMA provides core resources and assistance that help families navigate life with SMA. Families of SMA gives a stable, unbiased platform for families to live active, engaged and hopeful lives.   These resources include:
- Providing  SMA information to all newly diagnosed families helping each to understand and manage the disease better;
- Sending care packages of toys that have been recommended for SMA children;
- Housing a shared medical equipment pool which is available to families at no cost;
- SMA Community Connections; an online networking site for families across the globe to connect with one another;
- The Annual SMA Conference; the nation’s largest gathering of those affected by SMA and leading researchers from around the world.

Families affected by SMA turn to Families of Spinal Muscular Atrophy for information, guidance, encouragement and most importantly HOPE.

Through the continuous generosity of our families, volunteers and event participants, Families of SMA will remain steadfast in our quest to achieve the vision of our founders; a world where Spinal Muscular Atrophy is treatable and curable.

Our Specific Goals for 2011 Include:
FSMA has made significant progress in advancing new therapies for SMA, starting with funding the first ever SMA drug program in 2000. Now, the time is right for us to build on our success, broaden our research, and launch the next phase of therapeutic development work for SMA. FSMA is aiming to fund two new preclinical drug development programs in 2011, with more to follow. These multi-million dollar collaborations will focus on innovative methods of developing novel therapies for SMA, including both biologic and small molecule approaches.  Having multiple “shots on goal” gives us the best potential for reaching our objective of a treatment and cure for SMA.

In addition to progress on the research front, we have new opportunities that will improve medical care and the quality of life for SMA patients.  This new area of investment in patient care is the next logical step for our community and will build on the many support programs we already have for families, such as providing information and care packages sent to newly diagnosed families at no cost, as well as the FSMA equipment pool.  2011 also holds a little bit of magic for our community as The Annual SMA Conference will be held in Orlando, Florida at Walt Disney World.

Families of SMA funds and directs the leading SMA research programs. 
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 funded 5 leading new drug development programs for therapies specially designed to treat SMA.
-FSMA has invested significant resources into alternative approaches that show promise to cure SMA.

Click here to see our Fundraising Stars!

We are a non-profit, 501(c)3 tax exempt organization.  Federal ID# 36-3320440.

Click here to see our 990 tax forms.

Today we have 30 Chapters throughout the United States and over 70,000 members and supporters.  We are a collaborative organization where families and friends and researchers are all working together towards the same goal.

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.