Genetics & Its medicine’s
What is Genetics?
A disease caused by abnormalities in individual genome. Some examples are cystic
fibrosis, Falcille anemia, Marfan syndrome, Huntington and Hemochromatosis. Unique genetic disorders are genetically
informed in recognizable models: Autosomal dominates,
automatically diving and xlinked.
There are three types of genetic disorders:
• Distributed
disturbances, where mutations affect genes. An
example is sickle cell disease.
• Chromosomal disorders, in which chromosomes (or parts of
chromosomes) are missing or changed. ...
• Complex disorders in which there are mutations in two or more
genes.
Genetic Drugs
Cystagon (Cysteamine
Bitartrate)
Cysteamine that breaks down cystine in the body. It is
used to treat kidney cysts in children and adults. This medicine is used to help maintain kidney function and control kidney damage and other problems in
people who have an inherited condition that causes a certain natural substance
(cystine) to build up in the body (cystine) renal cyst).
(Form & Strength): 50mg, 150mg
Cerdelga (Eliglustat)
Cerdelga is a prescription drug used for the long-term treatment of adult Gaucher's disease type 1 (GD1). The ultrafast
metabolites of CYP2D6 may not be able to achieve sufficient concentrations of Serderga to produce a therapeutic effect. No specific dose is recommended for undetermined CYP2D6 metabolites.
(Form and strength): 84 mg orally once daily.
Zavesca
Zavesca is used to treat
mild to moderate type 1 Gaucher's disease in patients who do not receive enzyme replacement therapy.
Zavesca is indicated for oral treatment of adult patients with mild to moderate
type 1 Gaucher's disease.
Zavesca should only be used to treat patients who are not suitable for enzyme replacement therapy.
(Form and Strength): Capsule 100mg
Elaprase (Idursulfase)
Elaprase is a prescription drug for patients with Hunter's syndrome. Elaprase has been shown to
improve walking ability in patients over 5 years of age. Idursulfase contains natural enzymes that are deficient due to hereditary disorders. Eraplase helps replace this deficient enzyme. (Form and Strength): Inject 6 mg /
3 ml (2 mg / ml) into a
disposable vial.
Carbaglu
Carbaglu is a drug containing the active ingredient carglumic acid. It can be used as a dispersible tablet. "Dispersity" means that tablets can be dispersed
(mixed) in water.
Carbaglu is used to treat hyperammonemia (high levels of ammonia in the blood) in patients with the following
metabolic disorders:
N-acetylglutamate synthase (NAGS) deficiency.
Patients with this lifelong condition usually lack a liver enzyme called NAGS that helps break down ammonia.
If the enzyme is deficient, ammonia cannot be broken down and accumulates in the blood. Some organic acidemia (isovaleric acidemia, methylmalonic acidemia, and propionic acidemia) in which the patient
lacks certain enzymes involved in
protein metabolism.
(Form and strength): 200mg
tablet
Normosang (Human-Hemin)
Normosang helps to treat sudden attacks that occur in
patients suffering from acute liver porphyria. This
disease is characterized by liver collection of compounds (including porphyrin and its toxic precursors).
There are three types of liver porphila.
Acute intermittent porphyria,
• Porphyria variegata,
• Hereditary Coropropolia.
This accumulation results in the symptoms of the disease, including pain (mainly belly, backrest, thighs), nausea, vomiting and constipation.
(Form
and Strength): Normosang Injection 250MG
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