FDA establishes new advisory committee for genetic metabolic disease treatments

The FDA is as­sem­bling a new ad­vi­so­ry com­mit­tee to eval­u­ate po­ten­tial treat­ments for ge­net­ic meta­bol­ic dis­eases, the agency an­nounced Tues­day.

Ge­net­ic meta­bol­ic dis­eases are typ­i­cal­ly rare con­di­tions that dis­rupt a per­son’s me­tab­o­lism, the FDA ex­plained. The new pan­el will be com­posed of ex­perts in meta­bol­ic ge­net­ics, man­age­ment of in­born er­rors of me­tab­o­lism, small pop­u­la­tions, tri­al de­sign, trans­la­tion­al sci­ence, pe­di­atrics, epi­demi­ol­o­gy or sta­tis­tics and re­lat­ed spe­cial­ties.

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