As a funder of hC Bioscience, we’re happy to have them announcing their tRNA-based approach for targeting nonsense mutations at our annual Futures conference.
As we prepare to welcome families to the CureDuchenne FUTURES Annual Conference, scientific leaders, executives from nearly every pharmaceutical and biotech company focused on developing therapies for Duchenne and Becker […]
As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates in their next-generation exon skipping program for Duchenne amenable to skipping exon 51. In their […]
REGENXBIO provided a clinical and regulatory update on their Duchenne muscular dystrophy program highlighting progress in dose selection for their upcoming pivotal study which is expected to begin dosing patients […]
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The Duchenne muscular dystrophy community recently faced a profound loss—a young participant in Pfizer’s Phase 2 DAYLIGHT study (for boys 2 years to less than 4 years of age) passed away due to cardiac arrest, over a year after receiving an investigational micro-dystrophin gene therapy aimed at treating Duchenne.
Newport Beach, Calif. (May 7, 2024) – CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, announced its FUTURES National Conference will be held from May 23-26 in Orlando, Florida. FUTURES is a four-day event focused on bringing education, connection and hope to the Duchenne and Becker community. The annual event will take place as a hybrid event, offering attendees a chance to participate both online and in-person, at the JW Marriott Orlando, Grande Lakes.
Disney Parks can be great places for someone with a power wheelchair to enjoy the various characters roaming around the park, rides that you can drive your chair onto, and parades and performances.
We recently visited Disneyland with two Disney fans in power wheelchairs to see how it can be done. While it takes extra planning, strategy, and teamwork from friends, enjoying rides at Disneyland, including Rise of the Resistance, is possible even if you use a power wheelchair.
Our Duchenne journey began like so many others. Gabe, our fourth son, wasn’t reaching his developmental milestones. His doctor told us there was nothing wrong with him, and that, as the […]
