RALEIGH, N.C. (WNCN) – Parents of children with ultra-rare diseases are encouraging the Food and Drug Administration to act quickly to approve drugs that could treat their conditions and to take into account the limitations of clinical trials when considering drugs for rare and ultra-rare diseases.

Gilbert Dryden is a happy, seemingly healthy little boy.

"You look at this baby, he does not look like he is in heart failure; he does not look like he has skeletal weakness," noted Gilbert's mom, Madison Dryden. "He is developing on par with a typical 7-month-old."

In fact, Gilbert is living with an ultra-rare genetic condition that, experts say, kills nearly half of babies born with it before they turn five. When Gilbert was 16 days old, he was diagnosed with Barth Syndrome, a life-threaten

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