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Metabolic Disorders Resource Center
Drug Monographs
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NEWS & FEATURES
Cuvrior Gets FDA Approval for Wilson Disease
Tirzepatide Superior to Placebo in Weight Loss Study
Vijoice Gets Accelerated Approval for PIK3CA-Related Overgrowth Spectrum
Edsivo Gets Breakthrough Therapy Status for Vascular Ehlers-Danlos Syndrome
Fetal Exposure to Cannabis Linked to Adiposity in Childhood
Hyftor Approved for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
Elamipretide Gets Orphan Drug Status for Friedreich Ataxia
FDA Places Clinical Hold on Investigational Gene Therapy for Phenylketonuria
Generic Version of Cystadane Now Available
Eplontersen Gets Orphan Drug Status for Transthyretin-Mediated Amyloidosis
Semaglutide Superior to Liraglutide for Weight Loss
Long-Term Medication Usage After Bariatric Surgeries Compared
FDA Rejects Intranasal Carbetocin NDA for Prader-Willi Syndrome
FDA Extends Review Period for Gene Therapies for Beta-Thalassemia, Cerebral Adrenoleukodystrophy
FDA Requests Clinical Outcomes Data for Galactosemia Treatment
Generic Version of Carbaglu Now Available
Weight Loss Medication Wegovy in Short Supply
Gene Therapy Gets Priority Review for Cerebral Adrenoleukodystrophy
