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Metabolic Disorders Resource Center
Drug Monographs
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NEWS & FEATURES
Drug Shortage and Manufacturing Delays for Zemaira
Setmelanotide Gets Priority Review for Bardet-Biedl Syndrome and Alström Syndrome
October 2021: 5 Notable Drug Approvals
Two-Year Data Show Significant Weight Loss With Wegovy
Alvelestat Designated Orphan Drug for Alpha-1 Antitrypsin Deficiency
Novel CRISPR Therapy Gets Orphan Drug Status for Transthyretin Amyloidosis
Rethymic Approved for Pediatric Congenital Athymia
Novel Sodium Phenylbutyrate Formulation Under Review for Urea Cycle Disorders
Cipaglucosidase Alfa, Miglustat Under Review for Pompe Disease
MPR Weekly Dose Podcast Episode 92
FDA Places Clinical Hold on Investigational Gene Therapy for Phenylketonuria
Intraperitoneal Therapy Gets Orphan Drug Status for Hyperammonemia
FDA Grants Orphan Drug Status to Investigational Krabbe Disease Therapy
Novel Gene Therapy for Wilson Disease Gets Fast Track Status
Nexviazyme Approved for Late-Onset Pompe Disease
Tesomet Receives Orphan Drug Status for Hypothalamic Obesity
Bylvay Approved for Progressive Familial Intrahepatic Cholestasis
FDA to Review Vutrisiran for Polyneuropathy of Hereditary ATTR Amyloidosis
