02 Feb
  • By Alicia Clough
  • Cause in

CureDuchenne Cares Begins 6th Season of Family Workshops and Summit Series in Oklahoma City, Oklahoma

NEWPORT BEACH, Calif., Feb. 2, 2018 /PRNewswire/ — CureDuchenne, a nonprofit that funds research to find a cure for Duchenne muscular dystrophy, pioneered the community-based CureDuchenne Cares program which provides families, caregivers, and individuals living with Duchenne an interactive education and supportive program.  With support from biotech and pharmaceutical companies, CureDuchenne Cares brings to local communities critical resources that can positively […]

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13 Dec
  • By Alicia Clough
  • Cause in

Happy Holidays from PTC Therapeutics

We are sharing a holiday message and community update from PTC Therapeutics. Dear Duchenne Community, As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory process for Translarna™ (ataluren) with the FDA. Your voice as passionate advocates has been instrumental in this process. As you know, […]

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07 Dec
  • By Alicia Clough
  • Cause in

Phase 1b clinical research study of mini-dystrophin for DMD

CureDuchenne is proud that another one of our funding projects is entering clinical trial. Pfizer Inc. is planning a Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of PF-06939926 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD).  This clinical research study is designed to evaluate the safety and tolerability […]

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28 Nov
  • By Alicia Clough
  • Cause in

This #GivingTuesday, the Bill & Melinda Gates Foundation will Match Your Donation to CureDuchenne

Today, November 28, join CureDuchenne for #GivingTuesday, the global giving day that connects millions of people together to support and champion the causes they believe in by donating and fundraising on their behalf. Following the Thanksgiving holiday and the kickoff of the holiday shopping season, #GivingTuesday is meant to inspire people to give back to charities […]

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22 Nov
  • By Alicia Clough
  • Cause in

Even More to Be Thankful For This Thanksgiving

When Thanksgiving comes around each year, I’m reminded of how much we actually have to be thankful for. As a Duchenne parent, I am thankful every day for the moments I get to spend with my son Hawken. On Thanksgiving, I am thankful for all those who continue to fight alongside us for a cure […]

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08 Nov
  • By Alicia Clough
  • Cause in

CureDuchenne Ventures Seed Funding to Exonics Helped Advance CRISPR Research and Attract Series A Financing to Accelerate Development of Lead Gene Editing Program in Duchenne

Dr. Eric Olson to Provide CRISPR/Cas9 and Gene Therapy Update During November 16 Webinar   We are delighted that Exonics Therapeutics has closed a $40 million Series A financing with The Column Group (TCG) to advance its lead SingleCut CRISPR gene editing program in Duchenne muscular dystrophy and potentially other neuromuscular diseases.  CureDuchenne Ventures helped […]

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03 Nov
  • By Alicia Clough
  • Cause in

Sibling Saturday: Finding Inspiration in All Shapes and Sizes

No matter how long we live, the ones who will have known us the longest are our siblings.  For siblings Wil and Emily Cook, the time they have together is more precious knowing Duchenne muscular dystrophy is a critical factor in Wil’s life, and they make the most of it. Wil and Emily grew up […]

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27 Oct
  • By Alicia Clough
  • Cause in

Recap of PTC Therapeutics Community Conference Call Re: Ataluren

          PTC Therapeutics held a community conference today regarding Ataluren.  Stuart Peltz, President and CEO, PTC Therapeutics, Inc., welcomed and thanked the community as well as provided an overview of the dispute process. The call included a Q&A session with questions that were submitted to the company in advance as well […]

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25 Oct
  • By Alicia Clough
  • Cause in

PTC to File a Formal Dispute Resolution, Host Duchenne Community Call

Dear Duchenne Community, As you might have seen, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in the United States. This means that the FDA believes ataluren cannot be approved at this time. We are extremely disappointed and we strongly disagree with […]

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