Search results for "ORPHAN DRUG|Designated|Treatment of sickle cell disease." in comments (approximate match)
Class (Stereo):
CHEMICAL (ACHIRAL)
Mitapivat (AG-348; PKM2 activator 1020) is a novel, first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte py...
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Decitabine was first synthesized by Pliml and Sorm in the Institute of Organic Chemistry, Czechoslovak Academy of Sciences in 1964. Later, the drug was approved by FDA for the treatment of myelodysplastic syndromes in patients with cancer. Upon admin...
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Rifaximin is a structural analog of rifampin and a non-systemic, gastrointestinal site-specific antibiotic. Rifaximin acts by inhibiting bacterial ribonucleic acid (RNA) synthesis and contributes to restore intestinal microflora imbalance. It is FDA ...
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Sirolimus is the USAN-assigned generic name for the natural product rapamycin. Sirolimus is produced by a strain of Streptomyces hygroscopicus, isolated from a soil sample collected from Rapa Nui commonly known as Easter Island. Although sirolimus wa...
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Clotrimazole is an anti-fungal medicine indicated for the treatment of vaginal yeast infections and tinea. It can be used either in combination with other drugs (betamethasone dipropionate) or alone, in form of topical or vaginal cream. The drug exer...
5-(hydroxymethyl)-2-furaldehyde (5-Hydroxymethyl-2-furancarboxaldehyde, 5‐hydroxymethyl‐2‐furfural) is found in garden onion. It is obtainable from various carbohydrates. 5-Hydroxymethyl-2-furancarboxaldehyde is present in tomatoes, tobacco oil etc. ...