Inherited Metabolic Diseases Medication

Popular Inherited Metabolic Diseases Drugs

Popularity Drug Name Drug Class Price Price Trend
Enzyme Replacement Therapies 24,455
See Prices
ELIGLUSTAT is used for the long-term treatment of adults with Gaucher disease type I. Eliglustat is not used in certain people with Gaucher disease type 1. Your doctor will perform a test to make sure this medicine is right for you.
Enzyme Replacement Therapies 519
See Prices
SODIUM PHENYLBUTYRATE (sow dee um fen il bue ti rate) is used to manage the amount of ammonia in the blood of patients with urea cycle disorders. This medicine is not a cure.
Enzyme Replacement Therapies 1,850
See Prices
GALSULFASE is an enzyme replacement. It is used to treat the symptoms of mucopolysaccharidosis VI (also known as MPS VI or Maroteaux-Lamy syndrome). This medicine is not a cure.
Enzyme Replacement Therapies 866
See Prices
LARONIDASE is a drug that is used to replace an enzyme that is missing in patients with some forms of mucopolysaccharidosis I (MPS I). It is used to treat some of the symptoms of the disease. This medicine is not a cure.
Enzyme Replacement Therapies 3,029
See Prices
IDURSULFASE is used to replace an enzyme that is missing in patients with Hunter syndrome. It is not a cure.
Enzyme Replacement Therapies 5,463
See Prices
AGALSIDASE BETA is used to replace an enzyme that is missing in patients with Fabry disease. It is not a cure.
Enzyme Replacement Therapies 985
See Prices
Enzyme Replacement Therapies 1,189
See Prices
ELOSULFASE ALFA is an enzyme replacement. It is used to treat the symptoms of Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). This medicine is not a cure.
Enzyme Replacement Therapies 3,826
See Prices
GLYCEROL PHENYLBUTYRATE (gli ser ol fen il bue ti rate) is used to manage the amount of ammonia in the blood of patients with urea cycle disorders. This medicine is not a cure.
Enzyme Replacement Therapies 2,497
See Prices
NITISINONE slows the breakdown of tyrosine. It is used to treat a rare disease called hereditary tyrosinemia type I.
Enzyme Replacement Therapies N/A N/A See Prices
Enzyme Replacement Therapies 789
See Prices
Taliglucerase alfa is a man-made form of an enzyme that is missing in patients with Gaucher's disease. It is used to treat the symptoms of Gaucher's disease. It is not a cure.
Enzyme Replacement Therapies 763
See Prices
ALGLUCOSIDASE ALFA is a drug that is used to replace an enzyme that is missing in patients with Pompe disease. It is not a cure.
Enzyme Replacement Therapies 1,422
See Prices
Velaglucerase alfa is a man-made form of an enzyme that is missing in patients with Gaucher's disease. It is used to treat the symptoms of Gaucher's disease. It is not a cure.
Enzyme Replacement Therapies 867
See Prices
IMIGLUCERASE is a man-made form of an enzyme that is missing in patients with Gaucher's disease. It is used to treat the symptoms of Gaucher's disease. It is not a cure.

Note: Popularity is based on total prescriptions for the brand and generic versions of each drug, regardless of the condition being treated. Some drugs are prescribed for multiple conditions.

Recent News

Copyright ©2016 GoodRx, Inc.

GoodRx is not sponsored by or affiliated with any of the pharmacies identified in its price comparisons. All trademarks, brands, logos and copyright images are property of their respective owners and rights holders and are used solely to represent the products of these rights holders. This information is for informational purposes only and is not meant to be a substitute for professional medical advice, diagnosis or treatment. GoodRx is not offering advice, recommending or endorsing any specific prescription drug, pharmacy or other information on the site. GoodRx provides no warranty for any of the pricing data or other information. Please seek medical advice before starting, changing or terminating any medical treatment.