fabry disease การใช้

• The company is pursuing approval for its drug to treat Fabry disease.
• Fabry disease affects only about 5, 000 people in the world.
• Two of the drugs are designed to treat Fabry disease, a rare inherited disease.
• They are generally inherited in an autosomal recessive fashion, but notably Fabry disease is X-linked recessive.
• Patients with Fabry disease lack an elzqle known as alpha-galactosidase that breaks down certain body fats.
• It is awaiting FDA action on Replagal, its proposed treatment of Fabry disease, another rare disorder.
• This is a symptom of Fabry disease.
• There are about 5, 000 people in the world with Fabry disease, a deficiency of an enzyme.
• Fabry disease, Kleine Levin syndrome and congenital insensitivity to pain with anhidrosis were featured in the second season.
• Both have treatments for Fabry disease, and Genzyme has been successful with Cerezyme, its treatment for Gaucher's disease.
• Both Replagal and Genzyme's drug, Fabrazyme, consist of an enzyme that is lacked by patients with Fabry disease.
• Rare causes of aortic stenosis include Fabry disease, systemic lupus erythematosus, high blood uric acid levels, and infection.
• Yet it is not clear that TKT is using the controversial technique to produce its drug for Fabry disease.
• Lynda Leibowitz remembers the day more than two decades ago when her husband, Chaz, was diagnosed with Fabry disease.
• Fabry disease, which affects 5, 000 to 10, 000 people worldwide, clogs major organs such as the kidney and heart.
• The setback for TKT could help rival Genzyme General, which is also seeking approval of a drug for Fabry disease.
• Fabry disease is a rare inherited genetic fat-storage disorder caused by deficiency of an enzyme involved in the digestion of lipids.
• It is developing four more drugs, including Fabrazyme, a enzyme replacement drug for Fabry disease, which affects more than 2, 000 males.
• The main members of this group are Niemann Pick disease, Fabry disease, Krabbe disease, Gaucher disease, Tay Sachs disease and metachromatic leukodystrophy.
• Fabry disease is a rare, inherited disorder in which the body fails to produce an important enzyme, leading to pain and organ damage.