• Opinion: FDA needs to build in more flexibility for rare disease clinical trials

    25 days ago - By STAT

    My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease - he has the type known as CLN3 - is stealing his childhood. And then this rare disease will steal my child.
    Wheeler is missing the DNA needed to recycle a waste product called lipofuscin that his cells naturally produce. As lipofuscin builds up, his condition will get worse, robbing him of his eyesight, his speech, his mobility, and ultimately his life.
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