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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

Tolnaftate

Tolnaftate is a synthetic over-the-counter anti-fungal agent. It may come as a cream, powder, spray, or liquid aerosol, and is used to treat jock itch, athlete's foot and ringworm. It is sold under several brand names, most notably Tinac...

Piroxicam

A cyclooxygenase inhibiting, non-steroidal anti-inflammatory agent (NSAID) that is well established in treating rheumatoid arthritis and osteoarthritis and used for musculoskeletal disorders, dysmenorrhea, and postoperative pain. Its lon...

Hydroxyzine

Hydroxyzine is a first-generation histamine H1-receptor antagonist of the dephenylmethane and piperazine classes that exhibits sedative, anxiolytic, and antiemetic properties. It was first developed in 1955, and has since remained a rela...

Methotrexate

Methotrexate is a folate derivative that inhibits several enzymes responsible for nucleotide synthesis. This inhibition leads to suppression of inflammation as well as prevention of cell division. Because of these effects, methotrexate i...

Ulobetasol

Ulobetasol is a highly potent corticosteroid. It is structurally related to clobetasol. Due to its high potency, it is mainly prescribed in the treatment of severe plaque psoriasis and corticosteroid responsive dermatoses. Ulobetasol was...

Sumatriptan

Sumatriptan is a serotonin receptor agonist commonly used to treat migraines and sometimes cluster headaches. Sumatriptan is the first of the triptans and was made available in Europe in 1991 to treat migraines. Sumatriptan was granted F...