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Metabolic Disorders Resource Center
Drug Monographs
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NEWS & FEATURES
Skysona Gets Accelerated Approval for Early, Active Cerebral Adrenoleukodystrophy
Patient-Doctor Disagreements More Likely in Overweight Patients
FDA to Review Velmanase Alfa for Alpha-Mannosidosis
August 2022: Notable Drug Approvals
Orkambi Indication Expanded to Include More Children With Cystic Fibrosis
Oral Glycogen Synthase Inhibitor Designated Orphan Drug for Pompe Disease
Xenpozyme Approved for Acid Sphingomyelinase Deficiency
Drinking at Least 2 Cups of Tea Daily Linked to Reduced Mortality Risk
Hyftor Topical Gel Now Available for TSC-Associated Facial Angiofibroma
Time-Restricted Eating Early in Day More Effective for Weight Loss
Patisiran Promising for Transthyretin-Mediated Amyloidosis With Cardiomyopathy
USPSTF Still Says Individualize Decision for Diet, Exercise Counseling
Obesity Up in US Children, Teens From 2011 to 2020
June 2022: Notable Drug Approvals
Qsymia Approved for Chronic Weight Management in Pediatric Patients
Eplontersen Bests Placebo in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Study
Imcivree Approved for Weight Management in Bardet-Biedl Syndrome
Little Difference Seen in Weight Changes Before, After COVID-19 Shutdown
