Genetic Named patient medicines and treatment

 It is very difficult to manufacture, approve and release all drugs, especially for rare diseases. Patients with rare diseases often suffer from being unable to treat them. However, some drug consulting services (such as AlleviareIndia) provide services to doctors, hospitals, patients, and government hospitals who have designated patient drugs approved by the named patient program. Genetic diseases are such rare diseases, most of which have not been treated or received special treatment. However, by distributing required drugs and FDA-approved drugs, "AlleviareIndia" supports the use of designated patient supplies for treatment. Some of these drugs are discussed below:

    Zavesca Miglustat-used to treat mild to moderate type 1 Gaucher disease. It comes from both parents and is usually found in patients who lack the enzymes naturally present in the body and destroy the chemical substance called glucosylceramide. Therefore, when the enzyme is not produced, glucosylceramide will not be destroyed and cause spleen and liver enlargement, blood diseases and bone diseases. Zavesca Miglustat prevents the body from producing glycosylceramides, thereby minimizing discomfort.
    Elaprase Idursulfase-It can treat Hunter's syndrome that lacks enzymes, usually replaced by Elaprase Idursulfase. The disease never supports any enzyme replacement therapy, so there is no permanent cure.
    Cystine-needed to treat renal cysteine. Cystine reduces the accumulation of cysteine ​​in certain cells, thus delaying the development of renal failure.
    Cerdelga Miglustat-Like Zavesca Miglustat, Cerdelga has the same purpose, but is designated for long-term treatment of adult patients with Gaucher disease type 1.