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Glycogen storage disease type II (GSD-II), also called Pompe disease, and formerly known as GSD-IIa or Limb–girdle muscular dystrophy 2V, is an autosomal recessive metabolic disorder [1] which damages muscle and nerve cells throughout the body.
The volume of distribution of avalglucosidase alfa was 3.4 L in patients who had Pompe disease of a late onset. [12] The average half-life of avalglucosidase alfa was 1.6 hours, measured in patients with late stage Pompe disease. [12] There is little information available on the metabolism of the avalglucosidase alfa.
The FDA gives approval to Sanofi's (SNY) Nexviazyme (avalglucosidase alfa) for treating late-onset Pompe disease.
Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD). [6] Chemically, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha ...
The Federal Trade Commission had its first win earlier this week in its crusade to block drug company deals: Sanofi on Monday ended its up-to-$735 million licensing deal with Maze Therapeutics.It ...
Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). [4] [5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase ...
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