Inherited Metabolic Diseases Medications

BETAINE treats high levels of the amino acid homocysteine in your body (homocystinuria). Changes to diet are often combined with this medication.

LARONIDASE treats mucopolysaccharidosis I (MPS I). It works by replacing an enzyme normally made by the body, which reduces the buildup of complex sugars in the body. This decreases symptoms.

AGALSIDASE BETA is used to replace an enzyme that is missing in patients with Fabry disease. It is not a cure.

FOSDENOPTERIN treats molybdenum cofactor deficiency (MoCD). It works by replacing a substance normally made by the body, which reduces the buildup of toxins. This reduces injury to the brain.

IMIGLUCERASE treats Gaucher disease. It works by decreasing the amount of fatty substances (lipids) your body makes, which prevents the buildup of lipids in the spleen, liver, and other tissues. This decreases symptoms.

IDURSULFASE is used to replace an enzyme that is missing in patients with Hunter syndrome. It is not a cure.\n \nThis medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

PEGUNIGALSIDASE ALFA treats Fabry disease. It works by replacing an enzyme normally made by the body, which reduces the buildup fatty substances (lipids) in the eyes, kidneys, heart, and other tissues. This decreases symptoms and the risk of serious health problems.

ODEVIXIBAT treats itching caused by liver disease. It works by decreasing the amount of bile acids in the body, which reduces itching.

Aqneursa is part of the Amino Acid Derivatives class and treats Inherited Metabolic Diseases. Amino Acid Derivatives are used to treat a wide range of diseases, including inherited metabolic disorders, cardiovascular diseases, liver disorders, ulcers, and neurological diseases. They work by acting as signaling molecules that influence gene expression, thus regulating the rate at which specific proteins are synthesized within a cell. Aqneursa is only available as a brand name drug.

Ctexli is part of the Bile Acids class and treats Inherited Metabolic Diseases. Bile acids are used to dissolve gallstones and to treat cirrhosis by increasing the amount of ursodiol in the body. Ctexli is only available as a brand name drug.

Miplyffa is part of the Heat Shock Protein Amplifiers class and treats Inherited Metabolic Diseases. Heat Shock Protein Amplifiers are used to treat inherited metabolic diseases. They work by manipulating HSPs, which are proteins that help other proteins fold correctly. This helps cells survive stressful conditions. Miplyffa is only available as a brand name drug.

NEDOSIRAN treats high levels of the waste product oxalate in the urine, which can cause kidney stones and other problems. It works by decreasing the amount of oxalate your body makes.

Harliku is part of the HPPD Inhibitors class and treats Inherited Metabolic Diseases. HPPD inhibitors, also known as hydroxyphenyl-pyruvate dioxygenase inhibitors, are used to treat alkaptonuria. They work by preventing build up of HGA, homogentisic acid, in the body. Harliku is only available as a brand name drug.

MARALIXIBAT treats itching caused by liver disease. It works by decreasing the amount of bile acids in the body, which reduces itching.

LUMASIRAN lowers urinary oxalate levels in children and adults with primary hyperoxaluria type 1 (PH1).

Kebilidi is part of the Gene Therapy Drugs class and treats Inherited Metabolic Diseases. Gene therapy drugs are used to treat leukemia, blindness, and genetic diseases. They work by adding new genes to the body to compensate for defective ones. Kebilidi is only available as a brand name drug.

ELIVALDOGENE AUTOTEMCEL treats cerebral adrenoleukodystrophy (CALD). It works by helping your body make more of a protein needed to support the protective membrane that covers nerve cells in your brain and spinal cord. This delays worsening symptoms.

SODIUM PHENYLBUTYRATE treats urea cycle disorders (UCDs). It works by helping your body decrease the amount of ammonia in your blood. This lowers the risk of serious health problems. It is not a cure.

ELIGLUSTAT treats Gaucher disease. It works by decreasing the amount of fatty substances (lipids) your body makes, which prevents the buildup of lipids in the spleen, liver, and other tissues. This decreases symptoms.

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.\n \nThis medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

Forzinity is part of the Mitochondrial Cardiolipin Binders class and treats Inherited Metabolic Diseases. Mitochondrial Cardiolipin Binders are used to treat inherited metabolic disorders. They work by binding to cardioliptin in the mitochrondrial inner membrane, which improves mitochondrial structure and energy production. Forzinity is only available as a brand name drug.

SEBELIPASE ALFA treats lysosomal acid lipase deficiency (LAL-D). It works by replacing an enzyme normally made by the body, which reduces the buildup of fatty substances (lipids) in the body. This decreases symptoms.

ALGLUCOSIDASE ALFA treats Pompe disease. It works by replacing an enzyme normally made by the body, which reduces the buildup of sugar (glycogen) in your muscles. This decreases symptoms.

GALSULFASE is an enzyme replacement. It is used to treat the symptoms of mucopolysaccharidosis VI. This medicine is not a cure.\n \nThis medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

Nityr (nitisinone) slows the breakdown of tyrosine. It is used to treat a rare disease called hereditary tyrosinemia type 1. Nityr is less popular than comparable drugs. There are currently no generic alternatives for Nityr. It may be covered by Medicare and insurance plans, but some pharmacy coupons or cash prices may be lower.