Families of SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA.
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.
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 is explored in the quest for an effective SMA therapy. Families of SMA has funded more than 145 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 funded half of all the new Drug Development programs for SMA.
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. This includes investments to establish validated protocols for clinical assessment in every SMA type and to 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.
Our 2013 Goals: Recently, Families of SMA awarded seven basic research grants totaling $745,000 to develop new approaches for SMA therapies. We also made significant investments in more advanced programs including: The award of $750,000 to support preclinical development of a CNS-delivered Gene Replacement Therapy for SMA; and, The award of $700,000 for optimization of small molecule drug candidates which increase the SMA back-up gene to make them ready for human clinical trials.
Even with our current progress in adding new programs and advancing therapies to start clinical trials, we believe it is critical to do more. We are not slowing down, and we will continue to build upon our current momentum. Families of SMA is planning to award $1.4 Million in new funding over the next 6 months for the most promising research projects.
Families of SMA has been involved in funding half of the ongoing novel drug programs for SMA. We have been successful in implementing our model of early diversified investments followed by licensing programs to industrial partners for clinical development. Thank you for your support which has fueled this progress. We are also devoted to supporting families and patients by providing new Care Series booklets, sending newly diagnosed care packages, lending valuable equipment through the FSMA equipment pool, and hosting the Annual SMA Conference.
Today we have 31 Chapters throughout the United States and over 105,000 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.