Lysosomal Storage Disease Medications

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.

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.

CIPAGLUCOSIDASE 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.

CYSTEAMINE treats the buildup of cystine crystals in the eye. It works by decreasing the amount of the amino acid cystine in the eye. This prevents crystals from forming, which reduces eye pain and other symptoms.

VELMANASE ALFA treats alpha-mannosidosis. It works by replacing an enzyme normally made by the body, which reduces the buildup of complex sugars in the body. This decreases symptoms.

CYSTEAMINE treats the buildup of cystine crystals in the body. It works by decreasing the amount of the amino acid cystine in the body. This prevents crystals from forming, which prevents or delays worsening symptoms.

OLIPUDASE ALFA treats acid sphingomyelinase deficiency (ASMD). It works by replacing an enzyme normally made by the body, which reduces the buildup of a certain lipid (sphingomyelin) in your liver, spleen, and lungs. This decreases some symptoms of ASMD.

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.

AVALGLUCOSIDASE ALFA is an enzyme replacement therapy. It treats late-onset Pompe disease. It is not a cure for late-onset Pompe disease.

MIGLUSTAT 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.

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.

CYSTEAMINE treats the buildup of cystine crystals in the body. It works by decreasing the amount of the amino acid cystine in the body. This prevents crystals from forming, which prevents or delays worsening symptoms.

CYSTEAMINE treats the buildup of cystine crystals in the eye. It works by decreasing the amount of the amino acid cystine in the eye. This prevents crystals from forming, which reduces eye pain and other 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.

MIGALASTAT 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.

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.

VESTRONIDASE ALFA is an enzyme replacement. It is used to treat the symptoms of Mucopolysaccharidosis type VII (MPS VII; Sly syndrome). 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.

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.

MIGLUSTAT treats Pompe disease. It works by increasing the effects of cipaglucosidase alfa in the body to help it work better.

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

VELAGLUCERASE ALFA 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.

MIGLUSTAT 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.