The 2009 version of “The Good Fight”, Fight SMA’s Annual Spinal Muscular Atrophy Conference, wrapped up on Tuesday, April 28 and it was another successful year!

Here are a few of the highlights:

• 20 researchers and two additional graduate students from the National Institute of Neurological Disorders and Stroke (NINDS) were in attendance.

• Three of the attending scientists joined five additional doctors and healthcare professionals in an educational panel for attendees of the “Families and Friends Conference.”  This year’s panelists were Thomas Crawford, M.D., Tina Duong, MPT, Craig P. Eberson, M.D., Sally Evans, M.D., Sarah Feasel, MEd, RD, LD, CNSC, Mary Schroth, M.D., Kathy Swoboda, M.D., and Alex MacKenzie, M.D., Ph.D.

• On April 28, many of the families, friends and researchers descended on Capitol Hill to advocate for Spinal Muscular Atrophy and the SMA Treatment Acceleration Act, visiting the offices of more than 65 Congressional Representatives.  After a productive day, the SMA community was thrilled to learn that HR 2149, the SMA Treatment Acceleration Act, had been re-introduced in the House of Representatives.  FightSMA thanks all of our families, friends and researchers for kicking off the 2009 advocacy efforts in a big way!

• For the first year, Fight SMA offered a webinar of the Families and Friends panels.  The webinar was a success, with 30 participants from 13 U.S. states and a total of four countries.  Those who utilized the webinar said the experience was great!

We’re thankful for the contributions of everyone who made this conference a success, and for our sponsors, who made it possible: PTC Therapeutics, Genzyme, Hill-Rom Company, ISIS Pharmaceuticals, Psychogenics, Permobil, The Medi-Kid Co., and Exceptional Parent Magazine.  A special “thank you” goes to Hill-Rom Company and Genzyme, which not only sponsored the event, but also sent representatives to attend the Families and Friends Conference!

We are very pleased to announce that legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, H.R. 2149, the “SMA Treatment Acceleration Act of 2009“, has been introduced in the U.S. House of Representatives by Congressman Patrick Kennedy (D-RI) and Congressman Eric Cantor (R-VA).  On behalf of our organizations and the families affected by this deadly disease, we want to express our gratitude and thanks to Congressmen Kennedy and Cantor for their leadership.

This legislation builds upon the success of the SMA Treatment Acceleration Act introduced in the 110th Congress, which garnered 85 cosponsors in the House and 21 in the Senate, including then-Senator Barack Obama.  The new version of the legislation has been modified slightly to ensure resources for SMA clinical efforts and infrastructure are maximized.

SMA is the number one genetic killer of children under the age of two.  It is an inherited disease that destroys the nerves controlling muscle movement, which affects crawling, walking, head and neck control, swallowing, and breathing.  Approximately one in 40 people, or approximately 7.5 million Americans, carries the gene mutation that causes SMA.  Each child of two carriers of the mutant gene has a one in four chance of being afflicted by SMA.

Among more than 600 neurological disorders, SMA has been singled out by the National Institutes of Health (NIH) as one of the diseases closest to treatment based on scientists’ advanced genetic understanding of the disease and a strong collaboration between families, federal agencies, and patient advocacy groups.  Researchers have identified the gene responsible for SMA, as well as a disease modifying “back-up” gene that has opened the door to promising new treatment pathways.  This research is providing groundbreaking data for SMA and other disorders, including the muscular dystrophies, Lou Gehrig’s disease, Friedriech’s Ataxia, Fragile X syndrome, and Huntington’s disease.

In order to build on the progress being made by investigators and bring treatments to children affected by SMA, a broad coalition of organizations, including Families of SMA, FightSMA, the Muscular Dystrophy Association (MDA) and the SMA Foundation, has united behind the SMA Treatment Acceleration Act of 2009, legislation aimed primarily at supporting a national clinical trials network for SMA.

Specifically, the “SMA Treatment Acceleration Act of 2009” provides for the following:

• Federal support for a national clinical trials network for SMA;
• Federal support to enhance the SMA patient registry and for expanded research on the epidemiology of SMA;
• Establishes an Interagency SMA Research Coordinating Committee to include federal agencies including NIH, SMA researchers, and SMA families, to coordinate government activities relating to SMA, develop a comprehensive strategy for improving and expanding SMA research, make recommendations to strengthen collaborative research across multiple institutes at NIH, and identify barriers to the development of drugs for treating SMA; and
• Provides for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public

Our organizations are issuing a “Nationwide Call to Action” for all SMA families, researchers, and friends, to help engage every Member of Congress in support of this bill and the great efforts of Congressmen Kennedy and Cantor.

Kenneth Hobby
Families of SMA

Martha Slay
FightSMA

Annie Kennedy
MDA

Cynthia Joyce
SMA Foundation

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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

Click here to download the Joint Announcement.

The FightSMA Annual Conference: The Good Fight is off to a great start. Opening last night, attendees heard from Dr. Arthur Burghes of Ohio State University, Dr. Livio Pellizzoni of the Columbia University Center for Motor Neuron Biology and Disease, and Dr. Rashmi Kothary of Ottawa Health Research Institute.

The last speaker of the night was Dr. Clive Svendsen. Dr. Svendsen, his team at the University of Wisconsin-Madison, and Fight SMA Science Director Dr. Chris Lorson drew attention last year when they published the journal article “Induced pluripotent stem cells from a spinal muscular atrophy patient.” Work with induced pluripotent stem cells (IPS cells) and the potential promise that they hold for numerous diseases was so striking that Science declared it the 2008 Breakthrough of the Year .

It’s just a couple of days until the deadline to sign up for the free webcast of this year’s Fight SMA Annual Conference.  Response has been great!  In fact, it’s been international!  Here’s a rundown of who will be viewing the first ever webcast of “The Good Fight”, Fight SMA’s 2009 Annual Conference:

• Thus far, 31 people/families have signed up.  This means we have NINE spots left.
• The families signed up thus far represent 14 states in the U.S.: California, Florida, Kentucky, Massachusetts, Michigan, Missouri, Nevada, New Jersey, New Mexico, North Carolina, Ohio, Rhode Island, Utah, and Virginia.
• The webcast will reach a total of four continents!  In addition to the webcast participants in the U.S., families in Australia, Canada, India, and Romania have signed up.
• The webcast participants include adults and children with SMA Type 1, SMA Type 2, and SMA Type 3.

This webcast is helping the annual conference become what we’ve always hoped it would be — an educational opportunity that is available to everyone.

As mentioned previously, there are only nine spots left!  To take advantage of this FREE webcast, please contact Heather Lennon at Fight SMA via email by April 23!

The current economy, health issues, and other difficulties are keeping some from attending the 2009 FightSMA’s Families and Friends Annual Conference in Washington, DC. We are so sorry that these families cannot be with us, but we have some terrific news. This year we are pleased to offer remote access to a portion of the conference. On April 27th, the basic science update (5:00 - 6:00PM Eastern Time) and the Thriving with SMA Panel (6:30 - 8:15PM Eastern Time) will be webcast. Parents will be able to access the dynamic science and research update in real time from their home computers and join in the exchange with professionals who impact their children’s daily lives by chat room.

A limited number of spaces will be available for the webcast on a first come, first serve basis. If you would like to take advantage of this opportunity and sign up for one of the spaces, please email Heather Lennon by April 23rd.

For more information about the FightSMA Families and Friends Conference or about spinal muscular atrophy (SMA), please visit the FightSMA website at www.FightSMA.org.