CureDuchenne - Together we Can CureDuchenne
December 2014
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Message from Founder

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Debra and Hawken Miller

This year has been an eventful one for the Duchenne community. Every year we hope and pray this will be the year that we can say there is a cure for Duchenne. Each year we get closer to an approved treatment. Research and development takes time and has its ups and downs as you can see from this year’s research news.

Here are some research highlights from CureDuchenne funded projects in 2014:

  • Prosensa regained the rights of drisapersen from GSK and retains rights to all other programs
  • Sarepta Therapeutics announced plans for eteplirsen to submit NDA by the end of 2014
  • Akashi Therapeutics received fast track designation for HT-100 from FDA
  • CureDuchenne Ventures funds first social impact investment with Prosena
  • CureDuchenne announced $7 million collaboration with Prosensa to accelerate access to treatments for Duchenne
  • Dr. Kevin Flanigan from Nationwide Children’s Hospital published report with results of a study funded by CureDuchenne in Nature Medicine
  • PTC Therapeutics received conditional approval in the European Union for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy
  • Prosensa commenced re-dosing of drisapersen in North American in patients with Duchenne muscular dystrophy
  • Sarepta initiated Phase III clinical study for eteplirsen
  • Sarepta announced plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA
  • Prosensa began NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy
  • Prosensa extended the re-dosing of drisapersen in Europe in patients with Duchenne muscular dystrophy
  • Sarepta began dosing in confirmatory study of eteplirsen in ambulant patients with Duchenne muscular dystrophy
  • Sarepta initiated dosing in a clinical study of eteplirsen in non-ambulant patients with Duchenne muscular dystrophy
  • BioMarin acquired Prosensa and added Duchenne muscular dystrophy products to its rare disease portfolio

As the year comes to a close, Paul and I would like to thank all CureDuchenne supporters for your support over the years. We will continue to fund research until we find a cure for all boys with Duchenne.

The good news for boys with Duchenne is that there are potential treatments on the horizon. The bad news is this generation of boys will stop walking or die before they see the benefit of these drugs unless they can receive them soon. These boys cannot wait.

If you have donated to our year end campaign, we thank you for your generosity. Your contribution gives hope to the 300,000 boys worldwide with Duchenne and their families.

If you haven’t donated yet we could still use your support. Please go to: www.CureDuchenne.org/EOY2014.

Or mail a check to: CureDuchenne, 1400 Quail St. #110, Newport Beach, CA 92660. Donations in stock are also accepted.

Best wishes to you and your family for a joyous holiday season and a happy, healthy 2015.

With gratitude,

Debra Miller
Debra Miller
Founder and CEO
CureDuchenne

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