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Metabolic Disorders Resource Center
Drug Monographs
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NEWS & FEATURES
Novel Gene Therapy Granted Orphan Drug Status for Wilson Disease
Saxenda Approved for Obesity in Adolescent Patients
FDA Approves Imcivree for Rare Genetic Obesity Disorders
FDA Approves Oxlumo for Primary Hyperoxaluria Type 1
First Treatment for Progeria Gets FDA Approval
Semaglutide Ups Weight Loss When Added to Intensive Behavioral Therapy
Additional Palynziq Dosing Option Approved for PKU Treatment
Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked
Kalydeco Approval Expanded to Include Younger Cystic Fibrosis Patients
FDA Approves Once Daily Dosing Regimen for Nityr
FDA Cites Deficiencies in Veverimer NDA for Metabolic Acidosis in CKD
FDA Approves First Treatment for Long-Chain Fatty Acid Oxidation Disorders
CDC Updates List of Who Is at Highest Risk for COVID-19
Lumasiran Granted Priority Review for Primary Hyperoxaluria Type 1
Antiobesity Meds May Curb Weight Regain After RYGB
Lonafarnib Gets Priority Review for Progeria, Progeroid Laminopathies
Factors ID’d for Positive SARS-CoV-2 Test Result in Primary Care
Setmelanotide Granted Priority Review for Rare Genetic Obesity Disorders
