Fourteen years ago, I travelled to Russia with my family, just months after our son Hawken had been diagnosed with Duchenne muscular dystrophy. It was very raw for us at that time, we had no idea what to do, CureDuchenne was the furthest thing from our minds.  We were trying to deal with the diagnosis, […]
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  Exondys 51 is covered by STAR Kids, Texas’s Medicaid managed care program.   Type of information needed: Patient identification number Reference identification number Health plan Provider […]
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 our hearts long for. With the innovative CRISPR/Cas9 research being conducted by Dr. Eric Olson at the University of Texas Southwestern Medical Center, there is […]
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 Hawken has been, and always will be, the most meaningful role in my life. He has made me a proud father. His determination, kindness, and […]
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 a little more for their exemplary strength and courage as they battle side by side with their sons against this incurable disease. The boys put […]
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 a business environment to achieve life-changing treatments for patients. Such is the case for a promising new technology that has the potential to help 80% […]
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 his family. After stopping and starting the drug several times over the past few years, Alex once again has the distinction of being the first […]
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 will prioritize the patient and physician voice and experience. Rare diseases are complicated and regulators need to hear about the real world benefit that patients […]
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. For a 9-year old boy with Duchenne this was remarkable. For Doug’s parents this was a time of enormous hope. Created by Prosena, drisapersen was […]
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 editing and gene therapy to treat Duchenne muscular dystrophy. On Wednesday, May 10, the well-attended session entitled “Gene Therapies for Musculoskeletal Diseases was chaired by […]
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