If you live in Texas, are amendable to exon skipping 51, and you are having difficulty accessing Exondys 51, you or your provider may file a complaint to HPM_Complaints@hhsc.state.tx.us. Exondys […]
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 […]
Each year on Father’s Day, I like to take a moment and reflect on the memories I have with my family, specifically with my son Hawken. Being a father to […]
Father’s Day is the perfect time to reflect on all that our fathers do for us. For boys with Duchenne, it’s a day that they get to cherish their fathers […]
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 […]
