Ads
related to: pyruvate deficiency treatment medications side effects- Safety Data
Learn about the this pk deficiency
treatment's safety profile.
- Website For Patients
Visit this website if you are
a patient seeking information.
- Trials and Results
Learn about the first phase
3 pivotal trial program.
- Mechanism of Action
See how this pk deficiency
treatment works in your body.
- Safety Data
Search results
Results From The WOW.Com Content Network
The FDA approved mitapivat based on evidence from two clinical trials of 107 participants with pyruvate kinase deficiency. [3] Trial 1 (NCT03548220) of 80 adults with pyruvate kinase deficiency who did not receive regular blood transfusions and trial 2 (NCT03559699) of 27 adults with pyruvate kinase deficiency who received regular blood transfusions. [3]
Melarsoprol is a prodrug, a complex of melarsen oxide (a melamine derivative of phenylarsonous acid) with dimercaprol (also known as British anti-Lewisite, or BAL). It is metabolized to melarsen oxide in the body, which then acts by irreversibly binding to sulfhydryl groups on the enzyme pyruvate kinase, thus disrupting energy production in the parasite.
Pyruvate kinase deficiency is an inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cells. [ 4 ] [ 5 ] Both autosomal dominant and recessive inheritance have been observed with the disorder; classically, and more commonly, the inheritance is autosomal recessive .
Triheptanoin is used clinically in humans to treat inherited metabolic diseases, such as pyruvate carboxylase deficiency and carnitine palmitoyltransferase II deficiency. It also appears to increase the efficacy of the ketogenic diet as a treatment for epilepsy.
These side effects are serious and some of them are permanent, and many remain a crucial concern for companies and healthcare professionals and substantial efforts are being encouraged to reduce the potential risks for future antipsychotics through more clinical trials and drug development.
Mammals have two pyruvate kinase genes, PK-LR (which encodes for pyruvate kinase isozymes L and R) and PK-M (which encodes for pyruvate kinase isozyme M1), but only PKLR encodes for the red blood isozyme which effects pyruvate kinase deficiency. Over 250 PK-LR gene mutations have been identified and associated with pyruvate kinase deficiency.
Defective red cell metabolism (as in glucose-6-phosphate dehydrogenase deficiency and pyruvate kinase deficiency). [11] [12] Wilson's disease may infrequently present with hemolytic anemia without due to excessive inorganic copper in blood circulation, which destroys red blood cells (though the mechanism of hemolysis is still unclear). [13]
Pyruvate dehydrogenase deficiency (also known as pyruvate dehydrogenase complex deficiency or PDCD or PDH deficiency) is a rare neurodegenerative disorder associated with abnormal mitochondrial metabolism. PDCD is a genetic disease resulting from mutations in one of the components of the pyruvate dehydrogenase complex (PDC). [1]
Ad
related to: pyruvate deficiency treatment medications side effects