This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9. The second webinar in this series took place on Wednesday, September 6 at 2 PM ET, featuring Dr. Jerry Mendell of Nationwide Children’s Hospital who discussed…See More
Clinical research is going on Deletions. Anybody has any idea on Duplication's research.
Serepta Pharma working on 40-53 Deletions.
Does Gene Therapy is applicable for Duplication's as well. Please share .
Join us for a beautiful race series through downtown Victoria's Inner Harbor on October 8, 2017, and go the distance to end Duchenne!Click here to register or make a donation.Run For Our Sons is a program of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the U.S. focused entirely on Duchenne.See More
PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the country, featuring a roster of leading experts in the Duchenne space.You…See More
Sarepta Therapeutics, Inc. today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen (SRP-4053) in patients with confirmed deletions amenable to skipping exon 53. The study achieved statistical…See More
I'm sorry if i upset anyone with my message of any families with young boys, we experienced the most wonderful, fulfilling,caring, happy, joyous i could go on and on..of our lives with our darling boy he brought us so much pleasure we miss him so much but we will go on and carry on raising awareness and raising funds for the hospice (derian house) they were a fantastic support and still are enjoy everyday and live for every moment xxx
Thank you for replying to my message, its been the hardest few months of our lives, matthew was our world and we miss him like i cant explain, he was loved so much by all his family. he was such a well and happy boy, his passing came as such a shock it was so sudden and unexpected, we had had such a perfect day with him i still cant believe it happened and how we are coping without him with us. you read literature regarding the boys condition and me and my husband believed we were so lucky with matthew as he had always been in such good health, we were so lucky. when the boys reach a certain age things change for them and we didnt see it coming (something i will never forgive myself for) the care matthew received once in adult services wasnt the same or as through. thank you once again for replying its good to type away my thoughts xxx love lynn
Thank you so much! We are hoping to connect with others that are successfully finding ways to improve the quality of life for their sons.. and working to find a cure! I would be blessed by the information you mentioned. I'll take a look at that link. Thank you again for the welcome!