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 […]
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 […]
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 […]
CureDuchenne is pleased to see that the FDA will hold an Advisory Committee Meeting (AdCom) for PTC Therapeutics’s drug Ataluren. We are hopeful for a full and fair review which […]
For Doug Penner, now 14, being part of the original drisapersen trial was a hopeful time; the drug enabled him to go on four-mile walks, climb stairs and even jump. […]
The 2017 American Society of Gene & Cell Therapy took place in Washington DC last week. The Duchenne community was treated to a wealth of data from projects utilizing gene […]
PTC is committed to getting patients onto EMFLAZA as quickly as possible, without interruption of therapy. For patients that are currently accessing EMFLAZA through Master Pharmacy importation from outside of […]
Today, Dr. Eric Olson, PhD, Chair of Molecular Biology at UT Southwestern, was honored to give a presentation at the American Society of Gene & Cell Therapy (ASGCT) 2017 meeting […]
Today, PTC Therapeutics hosted a call for the Duchenne community to announce pricing and services for their launch of EMFLAZA, the commercial name for deflazacort. The announced price will be […]
