Carglumic acid is an orphan drug used for the treatment of hyperammonaemia in patients with N-acetylglutamate synthase deficiency. This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood-brain barrier and cause neurologic problems, cerebral edema, coma, and death.

Latanoprostene bunod is nitric oxide (NO)-donating prostaglandin F2a analog and is a topical ophthalmic therapeutic.

MALAT1-IN-1 modulated Malat1 downstream genes in a dose-dependent manner without affecting the expression of nuclear enriched abundant transcript 1 (Neat1).