23 Mar
  • By Alicia Clough
  • Cause in

What Is the Master Protocol for Duchenne?

Monday, I attended a meeting in Washington DC, hosted by Duke University’s Center for Health Policy, which included three sessions: Using Prior Data from Early Phase Trials to Inform Phase 3 Designs Utilizing Patient Registry and Natural History Study Data to Advance Therapeutic Development for Rare Diseases Leveraging Master Protocols for Trials with Small Patient […]

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14 Mar
  • By Alicia Clough
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Letter to the Duchenne community from Solid Biosciences about the status of the IGNITE DMD Clinical Trial

Today we announced that the U.S. Food and Drug Administration has placed our Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent. The patient was admitted to the hospital, received treatment and, as of the writing of this letter, is home with his family with no symptoms. Details about the event can be found in the press release we issued today, which is available here. The team at Solid will be working with the principal investigator and FDA to fully understand the cause and nature of this event, as well as identify appropriate next steps as soon as possible.

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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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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
  • 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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