Families of Spinal Muscular Atrophy Committed $2.5 Million to Funding SMA Research in 2012

ELK GROVE VILLAGE, Ill., Dec. 31, 2012 (GLOBE NEWSWIRE) -- Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, which includes: Basic Research to reveal the best ways of making SMA drugs; Drug Discovery to make practical new drugs, and; Clinical Trial resources to help test new drugs.

Families of SMA committed new funding to the following SMA research projects during 2012:
-$710,000 for Basic Research.
-$1,450,000 for Drug Discovery.
-$110,000 for Clinical Trial Resources.
-$200,000 for the 16th SMA Research Group Meeting.

Funding was provided to the following institutions: Northwestern University; Johns Hopkins University School of Medicine; University of Southern California; University of Ottawa; Indiana University; Nationwide Children's Hospital; CALIBR; The SMA Patient Registry; Ohio State University.

The basic research that Families of SMA has funded, through 145 research grants to 75 institutions around the world, has delivered major discoveries:
-The cause of SMA in now known. Which means that treatments can be developed that correct the underlying cause of the disease rather than just reduce symptoms.
-A back-up gene for SMA has been identified. Which means a straightforward drug target is already in the body: a built-in switch for new therapies to work on.

Using this knowledge, there are now have 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.

Families of SMA will announce more funding in the New Year, which will include 3 more new drug programs to add to the pipeline. In addition, the organization will launch a brand new effort for funding of patient care research in order to improve the standard of care.

About Families of SMA

Families of Spinal Muscular Atrophy (FSMA) is the world's leader focused on funding SMA research 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 SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization's work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provide straightforward targets for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients. For more information, visit: www.curesma.org.

Please contact Kenneth Hobby, President, at Kenneth@fsma.org or call (800) 886-1762 if you have any questions.

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Source: Families of SMA