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Archive for January, 2014

05
Jan 14

Looking back, looking ahead…research funded in 2013

We are thrilled to report we raised over $46,000 in 2013!  We were able to provide funding to four promising research projects. Here is a summary of projects we provided funding assistance in 2013:

  • Development of dystrophin independent therapy for DMD – a small molecule therapy using the zebrafish model to identify compounds that will compensate for the missing protein in boys with Duchenne;
  • Clinical Program to Evaluate HT-100 in DMD – development of  halofuginone hydrobromide, a delayed-release tablet for use in patients with DMD. In mdx mice and other disease models, halofuginone has been shown to halt and even reverse existing fibrosis (‘scar-like’ tissue), reduce inflammation, and actively promote regeneration of healthy muscle fibers;
  • Multicenter, randomized, double-blind, dose titration phase II study in patients with Duchenne muscular dystrophy to evaluate the efficacy and the tolerability of the combinations of Ibuprofen and Isosorbide Dinitrate in slowing Duchenne Muscular Dystrophy progression in non-ambulant patients;
  • Duchenne Superhighway – to identify inefficiencies in the drug approval process and streamline drug development.

 

This year, we plan to work even harder.  We are at a critical juncture–there is a drug progressing through clinical trials called Eteplirsen.  This drug has shown the most remarkable results of any drug tested to treat Duchenne, yet the FDA isn’t completely convinced it is ready for approval. As anyone living with this disease knows, time is not on our side and we must do everything we can to eliminate unnecessary hurdles that keep our boys from the drugs that will slow the progression of Duchenne, which Eteplirsen has demonstrated.

The Little Hercules Foundation is a member of the Duchenne Alliance, a 40+ member, international organization focused solely on moving promising research to trials in humans.  Our membership in the Duchenne Alliance allows us to engage directly with scientists and researchers conducting projects that may lead to a cure or treatment.

Stay tuned for what we have in store for you this year!  Save the date for our 2nd Annual 5K on July 12, 2014.  This year’s walk/race will be pet friendly, and we hope to see many of our four-legged friends racing for a cure right along with  us!

 

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