NOVEMBER 27, 2013
President Obama has SIGNED the NPRNA into law! We are incredibly excited about what this means for SMA research and our ability to reach a treatmentand/or a cure. THANK YOU to all of our friends who sent letters to their Representatives to help get this bill passed!!
For more into read the Q&A on FightSMA’s website to learn more about the bill, how it relates to SMA and what happens next visit: http://www.fightsma.org/blog/nprna/qa-the-national-pediatric-research-network-act-and-the-sma-community/
Chairman Upton commented, “At long last we are nearing the finish line, and we will soon have a network in place to help families like the Kennedys in Mattawan, Michigan, and their two young daughters who have the rare disease Spinal Muscular Atrophy. These two little angels, Brooke and Brielle, are little warriors in the effort to boost research for rare diseases and serve as an inspiration for all.” - See more at: http://energycommerce.house.gov/press-release/bill-help-children-rare-diseases-kennedy-girls-sw-michigan-now-one-step-closer-becoming-law#sthash.vh3lgboY.dpuf
MATTAWAN, MI – The medical struggles of two sisters from Mattawan helped inspire a bill funding research for rare diseases that passed the House by unanimous consent this week.
Brooke and Brielle Kennedy were born with type II spinal muscular atrophy. Their battle was chronicled in a 2012 Kalamazoo Gazette story.
The six-year-old and four-year-old have exceeded doctors’ prognoses, but SMA is a terminal, degenerative disease.
“SMA is the leading genetic killer of infants and young children. It is a terminal, degenerative progressive disease that results in the loss of nerves in the spinal cord and the weakness of the muscles connected with those nerves,” according to brookeandbrielle.com, a website set up by Sarah Kennedy, the girls’ mother. “SMA impacts the ability to walk, stand, sit, eat, breathe and even swallow.”
U.S. Rep. Fred Upton, R-St. Joseph, introduced the National Pediatric Research Network Act of 2013, which passed Tuesday as part of the Prematurity Research Expansion and Education for Mothers who Deliver Infants Early (PREEMIE) Reauthorization Act. Upton sponsored the original PREEMIE act, which became law in 2006.
The act supports research into rare and genetic diseases afflicting children, including SMA, by allowing the National Institutes of Health (NIH) to fund pediatric research consortium. Earlier this year, the measure passed the House as a stand-alone bill.
“This legislation will have a dramatic and life-changing effect on so many families who have suffered for so many years,” said Sarah Kennedy in a statement.
Upton said he met the Kennedys a few years ago, when they brought to his attention to a similar bill written to help fund rare diseases affecting children. Upton said he was charmed by the girls and keeps a picture of them in his Kalamazoo office.
“These girls are incredible. The first time I met Brielle and Brooke, they told me their names were Cinderella and Sleeping Beauty. They’re not letting their disabilities define them. But because spinal muscular atrophy is relatively rare, there hasn’t been a lot of funding for it,” said Upton in an email.
The bill will now move to the Senate as part of a package of health-related bills, S. 252. A previous version passed twice during the 112th Congress, once as a stand-alone bill and once as part of a larger piece of legislation. Neither version passed the Senate.
“The sad reality is it is often difficult to conduct research into rare diseases due to the small number of individuals with the disease,” he said. “We are working to change that and provide families with greater hope for a cure or advances in treatment.”
By Yvonne Zipp | email@example.com
on November 14, 2013 at 10:45 AM, updated November 14, 2013 at 2:31 PM. Yvonne Zipp is a staff writer at the Kalamazoo Gazette. Email her at firstname.lastname@example.org or follow her on Twitter.
Congressman Fred Upton’s official Press Release:
The Energy and Commerce Committee Press Release:
I'm beyond excited to share with you the BEST NEWS! The FDA has approved Gene Therapy for human clinical trials starting early next year. The trial will include newly diagnosed children 0-9 months old. If the trial is successful, the flood gates will open for more clinical trials and more children to be included and this could be the CURE for SMA.
Thank you Dr. Kaspar at Nationwide Children's Hospital and The Ohio State University for your tireless work and huge thank you to all of the SMA Foundations, Charities, families and friends that helped in any way to raise funds and awareness! We are so hopeful!
Earlier this year on a day when Brielle stayed home sick from school, we watched her school morning announcements online. Her elementary Principal talked about how received a letter from President Obama because he was given the award of “Outstanding Practicing Principal” by the Michigan Elementary and Middle School Principals Association. In the packet he opened from the White House one of the things he showed was a picture of Bo Obama, the presidential dog! Brielle decided she would like to draw a picture of Bo and write a letter to President Obama and tell him how much she aspires to be a Veterinarian when she grows up. She hoped to get a letter back some day. Just a couple months later - Brielle got a packet in the mail from the White House too!! The packet contained a letter from the President on official White House letterhead, a signed picture of President Obama, an interview fact sheet with the President (did you know when he was young, one of his favorite books was Where the Wild Things Are by Maurice Sendak?), a small picture of the dog Bo Obama and a really fun “Day in the life of Bo” pamphlet that details out Bo’s entire day. Bo is up at 6:30am and he heads off to bed around 7:00pm. His days sound very full of things to do! Brielle was overjoyed to receive this special packet in the mail. What a great way to learn about some American history.