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FDA approves 1st treatment for rare disorders in children, adults

from Healio

The FDA approved Cholbam, a cholic acid, for children and adults with bile acid synthesis disorders and peroxisomal disorders, according to a press release from the FDA. After the manufacturer was granted a rare pediatric disease priority review voucher, the FDA announced the approval of Cholbam as an oral treatment for adults and children at least three weeks old, according to the release. more


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