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Voxelotor

[L10403] and is unique from other drugs used to treat sickle cell anemia, such as [hydroxyurea], [L-glutamine], and [crizanlizumab][A188135,A188138] due to its novel mechanism of action.

Casimersen

Duchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive impairment of ambulatory, pulmonary, and

Fish liver oil

Copper histidinate

Menkes disease is a rare, X-linked recessive disorder caused by pathogenic variants in the ATP7A gene, which encodes a copper transport protein essential for absorbing dietary copper and transporting it

Dimenhydrinate

Dimehydrinate was first described in the literature in 1949, and patented in 1950. Early research into dimenhydrinate focused on its role as an antihistamine for urticaria; the treatment of motion sickness was an accidental discovery. Di...

Phenindione

An indandione that has been used as an anticoagulant. Phenindione has actions similar to warfarin, but it is now rarely employed because of its higher incidence of severe adverse effects. (From Martindale, The Extra Pharmacopoeia, 30th e...

Coagulation factor VIIa Recombinant Human

Recombinant human coagulation Factor VIIa (rFVIIa), intended for promoting hemostasis by activating the extrinsic pathway of the coagulation cascade. NovoSeven is a vitamin K-dependent glycoprotein consisting of 406 amino acid residues. ...

Noscapine

Pyrithione

Pyrithione zinc, or zinc pyrithione or zinc pyridinethione, is a coordination complex consisted of pyrithione ligands chelated to zinc (2+) ions via oxygen and sulfur centers. In the crystalline state, it exists as a centrosymmetric dime...

Desmopressin

It was designed through two primary chemical modifications of the natural hormone: the deamination of 1-cysteine and the substitution of L-arginine with D-arginine at the 8th position [A31661, A275458]