Italfarmaco released positive topline data from their Phase 3 trial in Givinostat, an anti-fibrotic for use in ambulant boys with Duchenne over 6 years old and on a chronic steroid […]
Significant decreases in key biomarkers of muscle damage.
Happy Father’s Day to all of the dads out there who have either found a way – or made one – so that their kids can experience their best life.
– Paul Miller, aka Hawken’s dad
Enjoy this hand-picked selection of books about Duchenne muscular dystrophy for children and parents. If you would like to add a book to this list, please let us know! Email: […]
Sarepta Therapeutics is currently running multiple clinical trials in a few different countries. They are at various stages and for a range of ages. Sarepta issued an update for the […]
CureDuchenne Ventures is pleased to share that one of our research investments, Code Bio, has announced that have secured $75 million in additional investments to advance their gene therapy programs […]
We’re thrilled to announce that researchers at Nationwide Children’s Hospital have documented the first-ever creation of full-length dystrophin in a human as a response to gene therapy. CureDuchenne has contributed […]
Lianna Orlando, PhD and Bradley Hodges, PhD Bring Scientific and Investment Expertise to Global Rare Disease Nonprofit’s Venture Philanthropy Team Newport Beach, Calif. – May 13, 2022 – CureDuchenne, a […]
