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Families of Spinal Muscular Atrophy Committed $2.5 Million to Funding SMA Research in 2012.
December 31, 2012.


Families of SMA committed new funding to the following SMA research projects during 2012:

$710,000 for Basic Research, announced in December 2012:
-$140,000 to Christine DiDonato, Ph.D. at Northwestern University.
-$140,000 to Charlotte Sumner, M.D. at Johns Hopkins University School of Medicine.
-$75,000 to Yong-Chao Ma, Ph.D. at Northwestern University.
-$75,000 to Chien-Ping Ko, Ph.D. at University of Southern California.
-$140,000 to Jocelyn Côté, Ph.D. at University of Ottawa.
-$140,000 to Sara Custer, Ph.D. at Indiana University.

$1,450,000 for Drug Discovery, announced in May 2012:
-$750,000 to Brian Kaspar, Ph.D. at Nationwide Children’s Hospital to advance a CNS delivered gene therapy for SMA.
-$700,000 to Peter Schultz, Ph.D. at CALIBR for a novel SMA drug program to optimize small molecule SMN enhancers.

$110,000 for Clinical Trial Resources, announced throughout 2012:
-$45,000 to The SMA Patient Registry to aid in clinical trial recruitment.
-$38,000 to Stephen J. Kolb, M.D. at Ohio State University for patient recruitment during the NINDS NeuroNext Biomarker Trial.
-$10,000 to Parent Project MD for a collaborative study exploring expectations in clinical trials.
-$17,000 to a Collaborative SMA Community Project to optimize clinical trial outcome scales.

$200,000 for the 16th SMA Research Group Meeting, held in June 2012:
-$200,000 for the largest meeting worldwide bringing over 225 scientists together to share new SMA research results and initiate new collaborations.

!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.

We now have 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.  Our 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. 

Your donation now will help us add to this momentum in 2013 so that we can accelerate the timeline to a treatment and cure.  We are getting ready now to announce more funding in the New Year, which will include 3 more new drug programs to add to the pipeline.   In addition, we are going to launch a brand new effort for funding of patient care research in order to improve the standard of care that our children receive.

Our research progress provides us all with hope that one day we will live in a world without SMA. Your gift will make that hope a reality. Click here or the image below to donate and support Families of SMA research and services.

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: www.curesma.org.


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