Genetic Named Patient Medicines and Treatment

Manufacturing, approving and launching all the drugs is difficult especially for rare diseases. Patients suffering from rare diseases commonly suffer as they remain devoid of treatment. However, some pharmaceutical consulting services like AlleviareIndia provide services to the doctors, hospitals, patients and government hospitals with named patient medicines approved under named patient programs.

Genetic ailments fall under such rare ones that stay mostly untreated or exceptionally treated. However, by the succor of distribution of the required and FDA approved medicines, ‘AlleviareIndia’ supports treatment with the named patient supplies of the same.

Some such medicines are discussed below:

● Zavesca Miglustat – It is used to treat mild to moderate type 1 Gaucher disease. It        comes from both the parents and is generally found in those patients who lack an enzyme which is naturally found in the body and breaks a chemical known as Glucosylceramide. Thus, when the enzyme is not produced, glucosylceramide is not broken and causes spleen and liver enlargement, blood disease and bone disease. Zavesca Miglustat stops the body from producing of glycosylceramide, which minimizes the discomfort up to a greater extent.

● Elaprase Idursulfase – It treats Hunter syndrome which lacks an enzyme from the body and is usually replaced by Elaprase Idursulfase. The disease never supports any enzyme replacement therapy and is therefore not permanently curable.

 Cystagon – Required for the treatment of Nephrophatic Cystinosi. Cysteaine reduces cysteine accumulation in some cells and thus delays the development of renal failure.

 Cerdelga Miglustat – Like Zavesca Miglustat, Cerdelga serves the same purpose, but is prescribed for long-term treatment of adult patients suffering from Type 1 Gaucher disease.

AlleviareIndia extends services for facilitation of such genetic diseases, their medicines and treatment.


More Posts

Send Us A Message