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The 200 commitments has an extremely simple, but critical goal:

To raise as a community the $1,000,000 needed to take the work of The SMA Gene Therapy program from Nationwide Children’s Hospital/Ohio State University out of the Lab and into the SMA Patient Population

This revolutionary research has the potential to cure Spinal Muscular Atrophy, as well as various other Neurological diseases. The earlier we can get into the SMA patient population, the greater the chances of rescuing the remaining motor neurons. The longer we wait, the greater the motor neuron loss in our SMA patient population. The time is NOW to stop the progression of the disease. Based on initial non-binding talks with the Food and Drug Administration there is a realistic timeline of what is necessary to ensure this program reaches human clinical trials. This program can reach Phase 1 in 12 months if the funding gap is closed. Based on Pre Pre IND talks with the FDA and the studies they have deemed necessary roughly $1,000,000 is needed.

We are asking for 200 commitments, every commitment is $5,000. We need 200 commitments within the next 12 months- for a total of $1,000,000 – with 100% of the proceeds going directly to fund Dr. Kaspar and the Gene Therapy program at Nationwide Children’s Hospital/OSU.

How you raise the money is up to you. Whether it is a run, a triathlon, a bake sale, a bowling event, a gala, a spaghetti fundraiser, the list of possibilities is endless. Make it fun, get your community involved. Do you know a company that is willing to sponsor a commitment? We will post all corporate sponsors on the front page for the entirety of this campaign! Can you raise $10,000? That would be amazing and would deduct 2 commitments from the goal! We welcome any and all other SMA organizations to join on board!

$1,000,000 seems like such a daunting task but it is a goal that is a achievable……. it will just take teamwork. We are not afraid of taking on a challenge so daunting……. we may not succeed, however we will try as hard as we can to make this trial a reality. Are you ready to become 1 of THE 200?

What is SMA?

  • SMA is the #1 genetic killer of children under the age of 2
  • SMA is estimated to occur in nearly 1 out of every 6,000 births.
  • 1 in every 40 people, or nearly 10 million Americans, unknowingly carries the gene responsible for SMA.
  • SMA is a pan-ethnic disease and does not discriminate based on race, ethnicity, or gender.
  • There is currently no treatment and no cure, but the National Institutes of Health (NIH) selected SMA as the disease closest to treatment of more than 600 neurological disorders.
  • Researchers estimate that a viable treatment and/or cure is attainable in as little as 5 years if provided the necessary funding.

 

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