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Happenings

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
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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