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 […]
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 the US, they may have immediate access to a “Bridge Program” that will provide EMFLAZA free of charge while their insurance status is being investigated. […]
Twenty years ago I was honored with the greatest gift I could have ever imagined, motherhood. As I think about the most precious gift in my life, my son Hawken, I am elated with happiness. Watching him grow up, his curiosity, big heart and sense of humor continue to inspire me and give me hope […]
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 in Washington DC for the George Stamatoyannopoulos Lecture series. Dr. Olson presented new research showing how gene editing technologies like CRISPR/Cas9 and Cpf-1 can be […]
At CureDuchenne we understand that when families are affected by a disease with no cure such as Duchenne muscular dystrophy, finding a sense of community is important. We are pleased to introduce our new CureDuchenne website, which is focused on strengthening the Duchenne community and connecting families to the best resources available. Our updated website […]
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