Guest blog by Tiffany Cook, CureDuchenne Family Resource Manager As a school-based Speech-Language Pathologist for more than 20 years and a Mom of a 17-year-old son with Duchenne muscular dystrophy, I can truly identify with the importance of conserving energy. For individuals with Duchenne muscular dystrophy, conserving energy involves physical energy as well as mental […]
CureDuchenne is proud to announce and welcome Tiffany Cook as its new Family Resource Manager. She, along with the CureDuchenne team, will be enhancing and presenting family education materials and investigating new ways to help families work with schools, insurance companies, clinicians, and other means of improving the lives of those families living with Duchenne. […]
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 HPM_Complaints@hhsc.state.tx.us. 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 […]