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.

We took many pictures during this year’s FightSMA Research Conference, held in Washington D.C on April 11 and 12. Below is a slideshow featuring some that we selected.  For a wrapup of this year’s conference, click here.

On April 11 and 12 , FightSMA and Spinal Muscular Atrophy researchers from around the world gathered for this year’s FightSMA Annual Research Conference in Washington, D.C.

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: