FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?
FDA Law
DECEMBER 13, 2023
Janet Maynard, Director of this Office, highlighted in the press release that “[g]enetic metabolic diseases include very rare diseases that individually affect a limited number of patients. Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”
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