On June 20th, Joshua’s Buddies, FightSMA’s chapter in Troy, Michigan, held its 10th Annual 5K Run/Walk/Roll to raise funds for Spinal Muscular Atrophy (SMA) research. Despite concerns about rain, crowds turned out to show their support of the fight against SMA - the #1 genetic killer of children under the age of two.

Thank you to the Cueter family (pictured below) and all their supporters for 10 great years!

Greetings SMA families, researchers and friends:

First and foremost, thank you for all you have done to help us increase awareness and attention to finding a treatment or cure for Spinal Muscular Atrophy (SMA). We are especially grateful to all of you who have taken the time to contact your Members of Congress in support of The SMA Treatment Acceleration Act of 2009 (H.R. 2149/S. 1158).

Thanks to our grassroots efforts, 17 Members of the House of Representatives and 4 Senators have now signed on to the SMA legislation. In order to keep this bill moving through the legislative process, we need to continue to grow the number of cosponsors of the bill. Our goal during the next two months is to encourage previous cosponsors from the 110th Congress to sign on to the bill again, while at the same time adding new members to the ranks. A list of our current cosponsors, as well as our target House and Senate cosponsors from the 110th Congress, are provided below. More than ever, we need your help! PLEASE contact your Members of Congress to ask them to cosponsor the SMA Treatment Acceleration Act.

Additionally, the July 4th recess which begins next Monday, June 29th is an ideal time for families to arrange meetings with their Senators and Representatives in their state and district offices. District meetings offer a unique opportunity in the middle of Congress’s long summer work period to focus Members’ attention on the SMA bill while they are away from the constant demands of their work on healthcare reform and other issues in Washington. PLEASE contact your Senators and Member of Congress’ state and district offices and request to meet with them over the July 4th recess.

As most of you are aware, the SMA Treatment Acceleration Act of 2009, introduced in the U.S. House of Representatives by Congressmen Patrick Kennedy (RI) and Eric Cantor (VA) and in the U.S. Senate by Senators Debbie Stabenow (MI) and Johnny Isakson (GA), provides federal support to complement the substantial private funding that is being invested to find a treatment for SMA. Passage of this landmark legislation will enable scientific investigators to mount national clinical trials and demonstrate that potential treatments are safe and effective for SMA patients.

THANK YOU AGAIN for your help for all those with SMA!

Sincerely,

NOTE: For more information on the “The SMA Treatment Acceleration Act” please contact any one of our Government Affairs staff:
Spencer Perlman, Families of SMA, spencer@fsma.org
Caroline Gibson, FightSMA, carolinegibson@fightsma.com
Annie Kennedy, MDA, akennedy@mdausa.org
Laura Lay, SMA Foundation, llay@wswdc.com

111th Congress Co-Sponsors
As of June 26, 2009

House of Representatives

Senate

To download this update, click here

Patrick’s Buddies’ 3rd Annual Rubber Duck Race was a tremendous success! Held Sunday, June 7, 2009 at Mindowaskin Park in Westfield, NJ, the race is estimated to have raised between $13,000 and $14,000 for spinal muscular atrophy (SMA) research. According to Maureen Higgins, head of the New Jersey chapter of FightSMA, the generosity shown by their supporters is extremely impressive given the charities in their area have been suffering during the economic downturn.

Recognition must be given to the employees of PTC Therapeutics for their tremendous support. Headquartered in South Plainfield, NJ, they had a “gaggleometer” in the office to tally their donations, which reached close to $1,000 from the individual employees! One of the PTC scientists who works on SMA, Amal Dakka, attended the event and helped Patrick give the signal to dump the ducks in the water at the start line. Maureen said, “I was very excited to have their support. It’s great that our supporters can see an actual scientists who benefits from this crazy duck race we throw every year!”

Grassroots efforts and work on Capitol Hill is fully underway to support the SMA Treatment Acceleration Act in the 111th Congress. The SMA community continues its combined collaborative efforts.

Taking advantage of its proximity to Washington, FightSMA visited Capitol Hill June 8th and 9th. Martha Slay, President of FightSMA, and Caroline Gibson, FightSMA Public Affairs Coordinator, traveled to Washington to advocate on behalf of the SMA Treatment Acceleration Act. They were able to meet with a number of health staffers and visited over 35 offices on Capitol Hill. Because of family constituent visits in April, meetings throughout the spring, and this recent trip to Capitol Hill, every Member of key committees has received materials and personal outreach regarding this paramount legislation for Spinal Muscular Atrophy research.

FightSMA and its partners are committed to the success of this first-ever SMA legislation, and plan to return to Capitol Hill throughout the 111th Congress.

Update to posting “Driving to cure spinal muscular atrophy“:

Newspapers and press outlets across the country have picked up on the story of the Strong’s journey. Even the Associate Press ran an article. Check out some of the news pieces by visiting The Gwendolyn Strong Foundation press page

Bill and Victoria Strong of Santa Barbara, California dove into the fight against Spinal Muscular Atrophy (SMA) after their daughter Gwendolyn was diagnosed with the most severe form of the disease, type 1. Not only did the Strong Family found the Gwendolyn Strong Foundation to raise awareness and research funding, but they started the Petition to Cure SMA. More than 64,800 people worldwide have signed the petition to show their support for the SMA Treatment Acceleration Act which was re-introduced in both the House of Representatives and the Senate.

The Strongs recently began a new project - turning a family reunion into an opportunity to raise awareness and funding for SMA research. The family will be driving from California to Mississippi, 5,000 miles round trip, so that Gwendolyn can meet her 104 year old great-grandfather. They are asking donors to Sponsor a Mile, with the proceeds going to support the Foundation’s mission.

The Strong’s and their campaign were profiled by Santa Barbara’s KEYT-3 news:

As it is the end of the school year, students across the country are graduating from their respective schools, including Katrina Gossett and Zak Schmoll. They are different ages, live in different cities, and were recently profiled by different newspapers. But they share at least two similarities: they both have spinal muscular atrophy (SMA) and they aren’t letting it hold them back.

Katrina just graduated with honors from the University of Chicago Law School. With her assistance dog Duke by her side, Katrina was the first wheelchair bound student to graduate from the school. During her three years at the University, she worked to make the school more accessible, in addition to her course study, work at a legal clinic, and participation in a law school musical. Click here to read the full article.

Zak will be speaking during his graduation ceremony as the salutatorian of his class. The seventeen year-old has been in a wheelchair since the age of 3 and despite enduring multiple surgeries to counteract the effects of SMA, Zak excelled academically. He hopes to turn that success into a business degree and possibly into a career in the business-side of sports. Click here to read the full article.

The Palmetto Scoop, a political blog in South Carolina, is reporting that SC Congressman Joe Wilson has signed on as a sponsor of the SMA Treatment Acceleration Act.  That brings the total number of sponsors in the House of Representatives to 16.  The bill would provide federal support to complement the substantial private funding that national non-profit organizations are investing to find a treatment for SMA. Passage of this landmark legislation would enable investigators to mount national clinical trials to demonstrate that identified therapeutics are safe and effective for SMA patients.

To read more about the proposed legislation and to download your own copy, please visit Fight SMA’s spinal muscular atrophy legislative information page.  To read more about Congressman Wilson joining the effort to pass the bill into law, please click here: Wilson sponsors important SMA legislation.

The 2009 Fight SMA Annual Conference featured a number of firsts, including the first-ever webcast of the event.  Now, the video from that webcast is available for online viewing and download!  You can watch it on the Conference 2009 page here at Fight SMA.  Just follow this link:

Fight SMA Annual Conference 2009

By Dr. Alex MacKenzie, Co-Chair of FightSMA’s Scientific Advisory Committee

It is with sadness we note the passing of Dr. Hung Li earlier this year. Dr. Li’s laboratory at Academia Sinica in Taiwan made a number of important and novel observations in the study of spinal muscular atrophy, all the more impressive given his was at a center with no prior record in SMA research. Dr. Li reported the first genetically and pathologically faithful murine model of severe SMA as well as the first report of treatment of these mice with an SMN2 inducing agent, butyrate. Latterly, he made the novel observation of a role for STAT5 kinase activation in the induction of SMN2, one of the first delineations of a mechanism of induction for this locus. As well he explored the role of apoptosis in SMA and reported on Valproate therapy in a small patient cohort.

In addition to his important SMA legacy, Dr. Li published widely on other issues including renal disease and novel therapies for stroke; clearly his was fertile and creative scientific mind. He was a quiet, kindly individual who reached out to a number of us with an invitation to Taiwan to visit his laboratory a number of years ago. A true pioneer in SMA research, he will be missed.