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Coping with Duchenne: Parents Swap Stories at ‘Futures’ Conference

Until her son, Will, was diagnosed with Duchenne muscular dystrophy as a 7-year-old, speech pathologist Tiffany Cook had never heard of the disease.

Today, it’s literally her full-time job.

As family resource manager with the nonprofit organization CureDuchenne, Cook — who works from her home in Dallas — helps other moms and dads of newly diagnosed Duchenne boys navigate individualized education programs (IEPs), among other challenges.

Dec 12, 2018
Clinical Trials and Research

Pathway Key to Muscle Regeneration Doesn’t Work as It Should in Duchenne, Study Finds

The Hippo signaling pathway — a key regulator of tissue development, and cell growth and death — does not work as it should in Duchenne muscular dystrophy and acts to prevent, rather than aid, the regeneration of muscle cells in DMD patients, a study reports.

A member of this pathway, a protein called YAP1, is an important regulator of muscle repair and regeneration after injury, and researchers suggest it could be a target for future Duchenne therapies. Interestingly, similar pathway dysregulation was not seen in other muscular dystrophies.

Nov 5, 2018