FDA needs to be more flexible assessing rare
/The FDA needs to be more flexible in assessing treatments forrare diseases, like the one that seemed to help my son
By Karen Quandt Sept. 7, 2022 Maria Fabrizio for STAT
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 atrial 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 humor, and — as we finally learned eight yearslater — a rare and fatal disease called Niemann-Pick type C. He appeared to be healthy at birth, and hit all of his first developmental milestones of walking and talking and growing. Yet we realized that he hadtrouble moving his eyes upward, and had to move his whole head to look up. Then his fine motor skills and balance began to lag, and he had difficulty learning. An enlarged spleen complicated the picture.Ty was eventually diagnosed with Niemann-Pick type C.
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