Hannah’s 2016: From Curling Toes to Gene Therapy

CaptureEleven-year-old Hannah Sames can still curl her toes, just barely. But time is running out. If Hannah can move her toes for a few more weeks, until she becomes the fourth child in a clinical

Understudy Gene Offers Hope for Spinal Muscular Atrophy

I began writing about genetics decades ago, and the best thing about getting older is witnessing the development of targeted treatments for single-gene diseases that I never thought would happen. But it is happening, for cystic fibrosis, Duchenne muscular dystrophy, … Continue reading »

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Catching Up With 3 Rare Disease Families

 

Four-year-old Eliza O’Neill’s viral videos, the subject of my last two blog posts, continue to dominate the news media with another appearance on The Today Show June 17. Hopefully, her family’s fight to fund gene therapy for her …

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Eliza’s Journey: Part 2

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Gene therapy is, finally, about to take off! The feeling was palpable at the American Society of Gene and Cell Therapy annual meeting a few weeks ago. It’s been nearly a quarter century since the first experiment in humans.

The drug …

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