rare diseases

9 Minutes

Time is running out for kids with a rare disease, parents say, unless the FDA acts

ustowner-013 2025-08-19 03:56

Gilbert Dryden probably only has enough medication to get him through the end of October, his mother, Madison, figures. Seven-month-old Gilbert has[……] ....

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5 Minutes

A Pennsylvania baby is the first to successfully receive personalized gene therapy

ustowner-013 2025-05-16 04:08

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment&nbsp[……] ....

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5 Minutes

The FDA is ignoring patients in clinical trials

ustowner-012 2023-05-30 03:14

Imagine having two daughters who both have a rare disease and watching them struggle — to be understood, to remember things, to learn, to be comfortab[……] ....

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