New Option Approved for Inherited Metabolic Disease That Affects the Brain

New Option Approved for Inherited Metabolic Disease That Affects the Brain

(MedPage Today) -- The FDA approved sepiapterin (Sephience) for the treatment of children and adults with phenylketonuria (PKU), PTC Therapeutics announced. The approval includes broad labeling for the treatment of hyperphenylalaninemia in adult...

FDA approves sepiapterin to treat phenylketonuria in adult, pediatric patients

“Without treatment, individuals with PKU would have very high blood Phe levels... so that results in neurocognitive deficits and mood disorders," Suzanne Hollander, MS, RD, LDN.

FDA Approves Sepiapterin for Treating Phenylketonuria in Adults and Children

The U.S. Food and Drug Administration (FDA) has approve […]

FDA Approves Sepiapterin for Adults, Children Living With Phenylketonuria

Sepiapterin treatment allowed for patients to liberalize their diet while reaching or exceeding recommended daily protein allowances in patients living with phenylketonuria.