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Metabolic Disorders Resource Center
Drug Monographs
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NEWS & FEATURES
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
FDA: More Data Needed to Support Arimoclomol in Niemann-Pick Disease Type C
Wegovy Approved for Chronic Weight Management
Tai Chi Helps Reduce Central Obesity in Adults 50 Years or Older
Synthetic Sepiapterin Gets Orphan Drug Status for Hyperphenylalaninemia
DDW: Semaglutide May Up Weight Loss After Endoscopic Sleeve Gastroplasty
Exercise, Liraglutide Combo Superior for Weight Loss Maintenance
Risk for Severe COVID-19 Increases With BMI Above 23
AGA Issues Guidelines on Intragastric Balloons for Managing Obesity
Nulibry Now Available for Molybdenum Cofactor Deficiency Type A
Sodium Bicarbonate May Mitigate Calcium Phosphate Stone Risk in CKD
