Help us drive to find a cure for Spinal Muscular Atrophy

Come participate in the 9th Annual Nicole Nadeau Golf Tournament Friday, June 25th, 2010

The Nicole Nadeau Golf Tournament is an annual tournament held in Southwest, Ohio to help raise awareness and funds for Spinal Muscular Atrophy (SMA). All proceeds from the outing will go directly to Families of SMA to fund research.

The event over the last eight years has raised over $106,000 for SMA Research!

What is SMA?

Spinal Muscular Atrophy (SMA) is the #1 genetic killer of children under the age of two.

Spinal Muscular Atrophy is an inherited disease that is often fatal. The disease destroys the nerves that control voluntary muscle movements like crawling, walking, shifting the head and neck and even swallowing. One in every 6,000 babies is born with SMA. Only 50% of the children under the age of two, who are found to have the disease, will live past their 2nd birthday.

Currently, there is no cure for SMA, but research efforts are making tremendous strides and a cure is within sight. With your support we can continue and hopefully accelerate research efforts to find a cure for this terrible disease.

"SMA not only has the kind of frequency and severity to warrant more research, but there are really good opportunities for developing a treatment.”

Dr. Kenneth H. Fischbeck
(Chief of neurogentics branch at the National Institute for Health) New York Times.

The search for a cure is progressing! Below are few of the numerous recently published press releases and articles regarding SMA research.

FSMA is funding three distinct drug discovery programs: 1) Quinazolines to boost SMN2 expression; 2) Tetracyclines at Paratek Pharmaceuticals to correct SMN2 splicing; 3) Motor neuron replacement program at California Stem Cell and UCI. These three programs represent an investment of over $16 Million so far in our pursuit to build a SMA drug pipeline.
FSMA awards 6 new basic research grants in 2009
Trophos (pharmaceutical company based in France) Increases Focus on Neuroprotection Including Spinal Muscular Atrophy. (December 2008)
Recent research pointed to a group of compounds called C5-quinazolines that showed promise for treating SMA (October 2008)
The SMA Treatment Acceleration Act”, has been introduced in the U.S. House of Representatives (August 2007)
Families of Spinal Muscular Atrophy Announces Major Breakthrough in Development of Drug Candidate (june 2007)
Families of Spinal Muscular Atrophy and Paratek Pharmaceuticals Expand Drug Discovery Collaboration for Spinal Muscular Atrophy (April 2007)
SMA CARNI-VAL TRIAL Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients with Spinal Muscular Atrophy (May 2005)
First UK Drugs Trials for SMA likely to start this year! (Feb 2005)
AmSMART enrolling children 0 through 2 years of age in an open label Rilutek clinical trial. Jan 2005)
FSMA awards 8 new research grants (for a total of 28 current grants) - Jan 11, 2005
FSMA partners with biotech firm to fight ailment drug giants avoid (Sep, 2004)

Families of SMA is 501(c)3 tax exempt organization that works to help individuals with SMA and their families. Families of SMA was founded in 1984 for the purpose of raising funds to promote research to find a cure for Spinal Muscular Atrophy and to support families affected by SMA.

Families of SMA is the largest private fund provider for SMA research and is leading the way to find a cure!

The ultimate goal at Families of SMA is to accelerate the discovery of an effective treatment and cure for SMA. To reach this end, our research program has three distinct parts:

basic research to unravel the biology of SMA,
pre-clinical drug discovery to develop SMA drugs, and
human clinical trial initiatives to test these new drugs

Families of SMA has provided more than $50 million in research funds to date, and have committed $15 million in new research funds over the next three years. FSMA is dedicated to funding research efforts, spending 88% of its budget on research.

FSMA also sponsors and organizes an Annual International SMA Research Group meeting, which is the largest conference that brings SMA research experts together, from around the world to collaborate, and the share the latest updates on SMA research.

The research efforts are making progress, but research is expensive. Through your generosity you can help continue this critical research and help find a cure to SMA once and for all.