House overwhelmingly passes bill to speed FDA drug approvals

A bipartisan bill that would make significant changes to the process for developing new drugs and medical devices overwhelmingly passed the House in a 344-77 vote Friday morning.

The bill, called 21st Century Cures, was cheered by rare across-the-aisle support from politicians, with 230 co-sponsors nearly evenly split between Democrats and Republicans. The pharmaceutical industry, patient advocacy groups, and medical organizations also support the bill, which calls for an additional $8.75 billion for the National Institutes of Health.

Michigan Senate Resolution No. 166 declares the entire month of August as SMA Awareness month in the state of Michigan!

Thank you Senator Tonya Schuitmaker for delivering Michigan Senate Resolution No. 166 to our house today! This resolution declares the entire month of August as SMA Awareness month in the state of Michigan!! It was adopted by the Senate on July 16, 2014.
The Resolution was offered by Senators Schuitmaker, Anderson, Bieda, Hildenbrand, Kowall and Proos. Thank you!! ‪#‎SMAawareness‬ ‪#‎curesma‬

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NORD Hosts "Portraits of Courage Celebration"

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It all started three years ago when we met with our Congressman to tell him about two special constituents in his district who have Spinal Muscular Atrophy and also to gain his support for the National Pediatric Research Network Act (NPRNA) - legislation created and championed by Fight SMA. Since then the NPRNA has passed Congress and has been signed into law by the President.

Chairman Fred Upton continues to push for accelerated cures in America by recently unveiling the 21st Century Cures initiative. The Energy and Commerce Committee released a video highlighting the merits of the program and cited the passage of the NPRNA as a model for Congress to use as it considers ways to support medical research in the future. The video can be found at http://bit.ly/about21stCC.

http://news.yahoo.com/nord-hosts-portraits-courage-celebration-160600330.html

Brooke and Brielle are "Decorated Little Generals" in the effort to boost research for rare diseases and they serve as an inspiration for every one of us! -Fred Upton

Chairman Upton is working on another bill - H.R. 2019 - The Gabriella Miller Kids Research Act of 2013.  This bill would prioritize funding for the research of pediatric rare diseases and disorders such as cancer, autism, fragile X and Spinal Muscular Atrophy.  It would eliminate political money and use the funds instead to expand the pediatric research through the National Institutes of Health’s common fund.
While talking on the House floor, Rep Upton mentioned how this bill would help his constituents —>Brooke and Brielle Kennedy and referred to them as “decorated little Generals” in the effort to boost research for rare diseases and they serve as an inspiration for every one of us!  

President Obama Signs NPRNA Into Law!!

BREAKING NEWS!

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

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Mattawan's Kennedy sisters help inspire House bill funding research into rare genetic diseases

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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 | yzipp@mlive.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 yzipp@mlive.com or follow her on Twitter.

http://www.mlive.com/news/kalamazoo/index.ssf/2013/11/mattawan_sisters_inspire_house.html

Congressman Fred Upton’s official Press Release:

Upton, House Approve Landmark Children’s Health Research Bill

The Energy and Commerce Committee Press Release:

Bill to Help Children with Rare Diseases Like the Kennedy Girls in SW Michigan Now One Step Closer to Becoming Law