25 Oct
  • By Alicia Clough
  • Cause in

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
  • Cause in

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
  • Cause in

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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21 Sep
  • By Alicia Clough
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#DuchenneChat hosted by CureDuchenne

CureDuchenne hosted a live Twitter chat on September 20 to raise awareness and address the important issues facing the Duchenne community. It was an opportunity to hear from patients, parents, caregivers and featured special guest Perry Shieh, MD, practicing clinician at the Center for Duchenne Muscular Dystrophy at UCLA, a certified Duchenne Care Center. Topics […]

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15 Sep
  • By Alicia Clough
  • Cause in

Join CureDuchenne for a live Twitter chat with special guest Dr. Perry Shieh from the Center for Duchenne Muscular Dystrophy at UCLA

CureDuchenne is hosting a Twitter chat #DuchenneChat on September 20 at 11 a.m. PDT/2 p.m. EDT to raise awareness and address the important issues facing the Duchenne community. This Twitter chat will be an opportunity to hear from patients, parents, caregivers and will feature our special guest Perry Shieh, MD, practicing clinician at the Center […]

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11 Sep
  • By Alicia Clough
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CureDuchenne has Funded More than $1 Million for Dr. Flanigan’s Duplication 2 Research. Here’s the Latest Research Update

Dr. Kevin Flanigan from Nationwide Children’s Hospital provided an update on his Dup2 research during a CureDuchenne hosted call with families on September 6. Back in 2010, Dr. Flanigan told me he was working on duplication mutations.  We knew so many families with these rare mutations, and at that time, the excitement seemed to be […]

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27 Jun
  • By Alicia Clough
  • Cause in

Collaboration to Fund CRISPR/Cas9 for Duchenne

Duchenne organizations are invited to fund CRISPR/Cas9 research at Exonics Therapeutics in a venture philanthropy collaboration. We are making progress on treatments for Duchenne, but a cure is the goal our hearts long for. With the innovative CRISPR/Cas9 research being conducted by Dr. Eric Olson at the University of Texas Southwestern Medical Center, there is […]

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13 Jun
  • By Alicia Clough
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Beyond the Lab – CureDuchenne Ventures Helps Accelerate Academic Research into Promising Medical Care for Patients

Academia has long been an incubating source of incredible medical breakthroughs. Sometimes it takes a little push and risk-taking to take that progressive research out of the lab and into a business environment to achieve life-changing treatments for patients. Such is the case for a promising new technology that has the potential to help 80% […]

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09 Jun
  • By Alicia Clough
  • Cause in

Meet Alex Chiabai – First Boy in Canada to Begin Drisapersen Redosing

Five years ago, 12-year-old Alex Chiabai was one of the first boy in Canada to begin taking drisapersen, a promising drug that provided a glimmer of hope for Alex and his family. After stopping and starting the drug several times over the past few years, Alex once again has the distinction of being the first […]

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