Analgesics
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Anesthetics
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Anti-Addiction/Substance Abuse Treatment Agents
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Antibacterials
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Anticonvulsants
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Antidementia Agents
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Antidepressants
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Antiemetics
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Antifungals
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Antigout Agents
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Antimigraine Agents
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Antimyasthenic Agents
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Antimycobacterials
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Antineoplastics
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Anti-Obesity Agents
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Antiparasitics
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Antiparkinson Agents
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Antipsychotics
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Antispasticity Agents
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Antivirals
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Anxiolytics
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Bipolar Agents
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Blood Glucose Regulators
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Blood Products and Modifiers
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Cardiovascular Agents
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Central Nervous System Agents
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Contraceptives
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Dental and Oral Agents
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Dermatological Agents
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Electrolytes/Minerals/Metals/Vitamins
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Gastrointestinal Agents
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Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
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Vosoritide
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Olipudase Alfa
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Alpha 1-proteinase Inhibitor, Human
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Agalsidase Beta
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Migalastat [DG:DG02691]
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Velmanase Alfa
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Cerliponase alfa
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Cholic Acid
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Cysteamine [DG:DG02658]
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Casimersen
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Eteplirsen
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Golodirsen
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Viltolarsen
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Alglucosidase Alfa
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Avalglucosidase Alfa
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Palovarotene
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Triheptanoin
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Imiglucerase
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Taliglucerase Alfa
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Velaglucerase Alfa
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Eliglustat [DG:DG00148]
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D09894 Eliglustat tartrate (JAN/USAN)
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Miglustat
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Uridine Triacetate
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Nitisinone
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Betaine [DG:DG00145]
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Idursulfase
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Lonafarnib
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Dichlorphenamide
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Asfotase Alfa
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Sebelipase Alfa
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Cromolyn [DG:DG00098]
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Setmelanotide [DG:DG03132]
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Fosdenopterin [DG:DG03102]
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Elosulfase Alfa
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Galsulfase
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Laronidase
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Vestronidase Alfa
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Pancrelipase
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Pegvaliase
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Sapropterin [DG:DG00147]
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Leniolisib [DG:DG02927]
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Afamelanotide [DG:DG02991]
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Givosiran [DG:DG03084]
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Lumasiran [DG:DG03131]
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Mitapivat
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Trofinetide
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Elapegademase
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Crizanlizumab
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Glutamine
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Hydroxyurea
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Voxelotor
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Nusinersen [DG:DG02002]
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Onasemnogene abeparvovec
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Risdiplam
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Sacrosidase
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Tafamidis [DG:DG01009]
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Vutrisiran [DG:DG03127]
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Glycerol Phenylbutyrate
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Obeticholic Acid
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Sodium Phenylbutyrate
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Belzutifan
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Burosumab
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Pegunigalsidase Alfa
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Inotersen [DG:DG02693]
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Patisiran [DG:DG02955]
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Genitourinary Agents
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Hormonal Agents, Stimulant/Replacement/Modifying (Adrenal)
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Hormonal Agents, Stimulant/Replacement/Modifying (Pituitary)
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Hormonal Agents, Stimulant/Replacement/Modifying (Prostaglandins)
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Hormonal Agents, Stimulant/Replacement/Modifying (Sex Hormones/Modifiers)
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Hormonal Agents, Stimulant/Replacement/Modifying (Thyroid)
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Hormonal Agents, Suppressant (Pituitary)
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Hormonal Agents, Suppressant (Thyroid)
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Immunological Agents
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Infertility Agents
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Inflammatory Bowel Disease Agents
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Metabolic Bone Disease Agents
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Ophthalmic Agents
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Otic Agents
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Respiratory Tract/Pulmonary Agents
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Sexual Disorder Agents
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Skeletal Muscle Relaxants
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Sleep Disorder Agents
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