The FDA needs to be more flexible in assessing treatments for rare diseases, like the one that seemed to help my son

Stat News

7 September 2022 - Every time I read about clinical trials testing possible treatments for rare diseases, I think of my son, Ty, whose brief but successful foray into such a trial highlights their value and their devastating limitations.

Ty was born in Pocatello, Idaho, in October 1996 with green eyes, a big personality, a great sense of humour, and — as we finally learned eight years later — a rare and fatal disease called Niemann-Pick type C.

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Michael Wonder

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Michael Wonder