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
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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
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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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08 Nov
  • By Alicia Clough
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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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27 Oct
  • By Alicia Clough
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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
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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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25 Oct
  • By Alicia Clough
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PTC Therapeutics Receives Complete Response Letter for Ataluren’s NDA

PTC Therapeutics received a Complete Response Letter from the Food and Drug Administration (FDA) for Ataluren’s New Drug Application (NDA).  This means that the FDA has completed its review of the drug application and has decided it will not approve the drug. We are deeply disappointed about the FDA’s decision about Ataluren.  We look forward […]

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06 Oct
  • By Alicia Clough
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CureDuchenne at World Muscle Society 2017

The 22nd International Annual Congress of the World Muscle Society took place in St Malo, between October 3 – 7, 2017. There were multiple breaking news stories on Duchenne muscular dystrophy research and development from academic groups and companies alike: Sarepta Therapeutics presented several posters on latest developments and breaking news which included: The effects […]

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03 Oct
  • By Alicia Clough
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Letter to the Community from PTC Therapeutics

Dear Duchenne Community, On behalf of all of PTC, we want to thank all of you for supporting the advisory committee review of ataluren. As you know, the meeting was held last Thursday, Sept. 28th. We were pleased that we could share the scientific data from our placebo controlled trials, our long-term extension studies, and […]

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28 Sep
  • By Alicia Clough
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CureDuchenne is funding Dr. Eric Olson’s research to develop CRISPR/Cas9 gene editing for Duchenne

Today, Dr. Eric Olson spoke at the UT Southwestern Wellstone Muscular Dystrophy Center Duchenne Muscular Dystrophy Clinical Symposium.  He presented promising pre-clinical data about gene editing using CRISPR/Cas9 for Duchenne. CureDuchenne is funding this research to advance Dr. Olson’s CRISPR gene editing development program that has the potential to correct 80% of Duchenne mutations. Early […]

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