Members of FightSMA’s Research and Legislative teams meet in Senate offices.

FightSMA continues to press for enactment of S.424/H.R.225, the National Pediatric Research Network Act (NPRNA), bipartisan legislation to support collaborative pediatric research including multi-site clinical trials for rare pediatric disorders like SMA.

Now that the bill has passed the U.S. House of Representatives, we are working with our Senate sponsors, Senators Sherrod Brown (D-OH) and Roger Wicker (R-MS), and a number of allied organizations to advance S.424 in the Senate Health, Education, Labor and Pensions (HELP) Committee.  Along with the Coalition for Pediatric Medical Research (CPMR), a group of more than 20 children’s hospitals around the country, we have held several very positive meetings with HELP committee staff on both sides of the aisle.

FightSMA scientists visited with health staff for numerous Senate HELP committee members during our annual research conference in April, and CPMR scientists provided a briefing for committee staff on May 7  to discuss the benefits of collaborative research networks.  We are proud to report that, with your help, committee members Johnny Isakson (R-GA) and Orrin Hatch (R-UT) have signed on to the bill, joining original cosponsor and fellow committee member Sheldon Whitehouse (D-RI).

Our goal now is to build additional support for the bill among Republican HELP committee members in order to position the NPRNA for a committee markup and approval in the near future. FightSMA needs your help in this effort.

A phone call, letter or email to the following Senators can make all the difference: 

Lamar Alexander (Tennessee)

Michael Enzi (Wyoming)

Richard Burr (North Carolina)

Rand Paul (Kentucky)

Pat Roberts (Kansas)

Lisa Murkowski (Alaska)

Mark Kirk (Illinois)

Tim Scott (South Carolina)

If you or anyone you know lives or works in the above states please make your voice heard and encourage others to do the same!  The support of families across the country has brought legislation to benefit SMA clinical trials closer to the president’s desk than it has ever been.  With your help, we can push the NPRNA over the finish line!

Thank you!

Find full contact information for your US Senators

Review our letter template and talking points for a call

$37,000…THAT is the astounding amount Party With A Purpose generated for SMA research! Thanks just doesn’t seem like enough as I am feeling so blessed and grateful to have so many wonderful friends & family who supported our efforts! Thank you to all who made a donation, attended the event, became a corporate sponsor, gave a donation for our raffle or those who went out there and got donations from their friends and family…wow! I believe my cousin said it best when she shared this quote from Margaret Mead…”Never doubt that a small group of thoughtful, committed citizens can change the world; indeed, it’s the only thing that ever has.” Thank you from my family, FightSMA and all those fighting to beat SMA!

Party With A Purpose 2013 was held at the Virginia War Memorial in Richmond, VA, on May 17. Over 140 attendees enjoyed the beautiful evening outdoors, with entertainment provided by DJ Bill “Evryday” Holliday. A highlight of the evening was the raffle, which included 40 amazing prize packages, all donated by area businesses. FightSMA extends heartfelt thanks all those in the Richmond/Hanover County community who continue to support our fight to end spinal muscular atrophy.

Carice and Andrew

Written by Carice Creighton Smith, head of FightSMA Hanover and this month’s Party with a Purpose 2013.

Every day, kids with SMA fight a fierce battle just to live. Spinal Muscular Atrophy (SMA) kills more young children than ANY other genetic disease. It’s an under-funded, incurable neuromuscular disease that robs children the ability to walk, talk, swallow, eat, and some, to even breathe on their own. While the mind remains unaffected, the muscles waste away. 1 in every 40 people carry this gene, unknowingly. Due to the lack of federal funding and the awareness it needs and deserves, the task has been left to the families and friends of these amazing, inspirational fighters, to GET the funding & awareness so desperately needed.

My son has been fighting SMA since he was born. And what a fighter he is! Continuing to beat the prognosis that he would not live past the age of 8, Andrew will turn 21 this September. Since 1998, our family, along with friends, has been supporting the national organization FightSMA by coordinating events for our chapter, FightSMA Hanover, “To strategically accelerate the search for a treatment and cure.”

With continued support, it has been amazing to see the awareness and attention that has been brought to SMA! SMA-specific drugs have already entered clinical trials. We fund researchers across the globe. The National Pediatric Research Network Act (NPRNA), which specifically addresses SMA and other rare, life-threatening pediatric diseases, has been passed in the House and is currently generating support in the Senate. Significant strides have been made in the care of kids living with SMA, giving clinicians and parents the tools and resources they need to care for their children and give them a “fighting chance!”

FightSMA is truly making a difference. But the road is long. Every day, a child is diagnosed with SMA. Every day, a parent is told to take their child home and, “enjoy the time time they have.” So we continue fighting!

On behalf of my family and all children & families who bravely endure this terrible disease and in memory of those who have lost the battle, thank you! Until no parent has to count their child’s life in months…sometimes days, we will continue fighting and I hope you will join us!

RVSP here for Party with a Purpose ! Or if you can’t attend, show your support by making an online donation.

The conference brought together leading SMA researchers and clinicians to review the year’s work in SMA science.  Nearly 40 dedicated scientists convened in Washington, D.C., for two days of intensive reporting, discussions, and collaboration–known as the hallmark of our scientific meetings. We are left inspired by the numerous developments and grateful to all those who are helping to make the path forward possible.

On April 11, five grantees were selected as recipients of the 2013 Emerging Investigator Awards, a program funded jointly by FightSMA and the Gwendolyn Strong Foundation. Now in its first year, the EIA program seeks to cultivate exploration and distinguish new talent within the SMA field. We were overwhelmed by the wealth of excellent EIA applications and are heartened by the great potential this identifies for SMA science. 2013 EIA winners, L-R: Dr. Constantin d’Ydewalle of Johns Hopkins University; Dr. Kevin Foust of the Ohio State University; Dr. Lyndsay Murray of Ottawa Hospital Research Institute; Dr. Hansjorg Rindt of University of Missouri; Dr. Steven Crone of Cincinnati Children’s Hospital Medical Center.

On April 12, FightSMA held its Annual Webcast, inviting families around the world to participate and interact live with a panel of SMA experts. On our panel this year were Dr. Brian Kaspar, Dr. Rashmi Kothary (pictured L-R), Dr. Kathy Swoboda, Dr. Christine DiDonato, Dr. Chris Lorson, and Dr. Alex MacKenzie.  You can view a recorded version of the webcast to the left, or view it on YouTube.

Finally, FightSMA maintained its strong presence on Capitol Hill last week by sending our scientists and representatives to advocate for SMA. Our team met with several Senate offices to affirm their endorsement of the National Pediatric Research Network Act of 2013. The NPRNA, developed in part by FightSMA, seeks to establish a network of clinical trial sites focused on rare diseases like SMA. (L-R: Dr. DiDonato; Steve Eichenauer, Public Strategies Washington; Dr. Lorson.)

Even if you can’t make it to Washington, you can still help this crucial legislation reach the President’s desk. Click here to read how.

FightSMA would like to extend its sincerest thanks to all of our 2013 corporate sponsors:

The Emerging Investigator Awards are designed to provide initial support for an outstanding idea from a junior investigator. This type of seed money can be particularly difficult to obtain in today’s climate, and the EI Awards will help spark transformative ideas into reality.

During the program’s first year, FightSMA and GSF received 24 excellent applications from across the globe (US, Canada, Spain, Italy, Netherlands, and the UK.) Ultimately, five outstanding candidates were identified and were announced during this weekend’s FightSMA Annual Research Conference in Washington, D.C.  Here are images of the grantees and details about their research:

	Dr. Lyndsay Murray of Ottawa Hospital Research Institute Program title: Development of Rho-Kinase inhibitors as a therapeutic for SMA Dr. Murray’s research focuses on understanding the basic biology of spinal muscular atrophy and in the development of Smn independent therapeutics. This project specifically looks at how modulation of the pathways which control actin dynamics can influence disease progression in mouse models of SMA.
	Dr. Steven Crone of Cincinnati Children’s Hospital Medical Center Program title: A circuit mechanism of degeneration in mouse models of SMA The goal of Dr. Crone’s research is to understand how the spinal circuitry that controls motor neurons is disrupted in SMA and to determine how disrupted motor circuitry contributes to pathology of SMA.
	Dr. Constantin d’Ydewalle of Johns Hopkins University Program title: Long non-coding RNAs as new therapeutic targets for spinal muscular atrophy Dr. d’Ydewalle is investigating how long non-protein-coding RNAs (lncRNAs) control SMN expression. To this end, he is using patient-derived cell lines as well as the SMA mouse model. He hypothesizes that interfering with the expression of lncRNAs increases SMN expression. Eventually, he believes that lncRNAs represent an innovative and promising therapeutic target for SMA.
	Dr. Kevin Foust of the Ohio State University Program title: Investigating Enteric Nervous System Function in Spinal Muscular Atrophy Dr. Foust is investigating the gastrointestinal function in two new models of SMA. He wants to understand the role of SMN within the neurons that control GI motility.
	Dr. Hansjorg Rindt of University of Missouri Program title: Astrocytes as novel determinant of disease progression in spinal muscular atrophy Astrocytes are cells in the central nervous system that supply neurons with growth factors and energy. In SMA, this function of astrocytes is disrupted, and this may affect the activity and survival of motor neurons in the spinal cord. Dr. Rindt’s research is aimed at restoring proper astrocyte activity which in turn will have a positive effect on the function of motor neurons.

Congratulations to our five grantees!  Each of them receives $25,000 for their work, and after six months, they will compete for two $62,500 grants that would cover one-year research projects.

For more information, read our news release on the EI Awards.  For more information on the FightSMA Annual Research Conference, including the annual webcast, click here.

The 14th Annual Hannah’s Buddies Charity Classic, as with previous years, featured a golf tournament followed by a concert and silent auction.  But, that description doesn’t fit what a great event this truly is.  It began in 2000 with John Bell of the band Widespread Panic deciding to help his goddaughter, Hannah, and tens of thousands of children in their fight against SMA. Today, the golf tournament, silent/auction and concert lineup attracts more than 2,000 participants and has raised nearly $2 million for SMA research.

The golf tourney was held on March 22 at the Grand Cypress Golf Club in Orlando.  The concert, dinner and silent auction, were the next day at the House of Blues in Orlando.  The concert, as always, featured John Bell.  Also on the list of acts were Nichol & The Polar Bears, Futurebirds and Wet Willie.

We continue to be amazed at the success of this annual event and we are extremely thankful and grateful for the support.  Below is a slideshow of some of the pictures from the event.  You can also see a wonderful video Hannah’s Buddies posted on Facebook by going here.

We’re very excited today to announce details of the FightSMA Webcast 2013!

Once again, SMA families from around the world will have the opportunity to hear some of the top SMA researchers discuss the state of the search for a treatment or a cure and have their questions answered by those experts! For the third year in a row, FightSMA is webcasting a portion of the annual research conference in Washington, DC, and you’re welcome to join in.

Details:
FightSMA Annual Webcast
Friday, April 12, 2013
6:30 p.m. EDT

There are THREE ways to participate this year!  You can tweet @FightSMA with the hashtag #SMAWebcast, email your question toweb@fightsma.org, or join in via our website at the time of the webcast. A link to join the webcast will be posted on the FightSMA home page on April 11.

Meet the FightSMA Webcast 2013 Panelists:

Click here to keep up with the latest details about the 2013 FightSMA Annual Research Conference

If you haven’t had the time to “like” us on Facebook, here’s a link: FightSMA on Facebook.

Introducing the NPRNA on Rare Disease Day on Capitol Hill, Senators Sherrod Brown (D-OH) and Roger Wicker (R-MS) continue their leadership from the 112th Congress by once again serving as the bill’s lead sponsors. Joining them as original cosponsors will be Senators Richard Blumenthal (D-CT), Roy Blunt (R-MO), Susan Collins (R-ME), Rob Portman (R-OH) and Sheldon Whitehouse (D-RI). The NPRNA will now be referred to the Senate Health, Education, Labor and Pensions (HELP) committee, where it is expected to be considered by committee members in the coming weeks. FightSMA and the Gwendolyn Strong Foundation, two of the bills’ endorsing organizations, are tremendously grateful to Senators Brown and Wicker and all of our supporters in the Senate for their tireless efforts, which give hope to thousands of families across the nation.

As this critical legislation is considered in the Senate, FightSMA needs your help. A phone call, letter or email to your senators can make all the difference. Your work during the 112th Congress brought the legislation closer to the president’s desk than it has ever been. With your help, we can push the NPRNA over the finish line!

Thank you so much for all that you do.