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 […]
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 […]
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 […]
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 […]
Leading a healthy lifestyle is something that any individual should aim towards. For those with Duchenne, making nutritional modifications has the potential to improve their overall health. Research has indicated that proper nutrition is one component that can improve the overall quality of life for a person living with Duchenne.     Seven Daily Points […]
CureDuchenne firmly believes in working together to help any child with Duchenne muscular dystrophy find the support they need. Duchenne is a disorder which knows no cultural, economic, or social boundaries.  This fatal genetic muscle disease affects more than 300,000 children worldwide. Especially affected are muscular dystrophy patients in Nepal, as the region lacks resources […]
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 […]
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 […]
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 […]
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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