Opinion: My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility

It's time for @DrCaliff_FDA and the FDA to fundamentally reimagine how drugs for rare diseases are developed and remove needless obstacles that hinder clinical trials for them.

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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