<unk> , sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and the first oral medication approved to treat this disease.<unk>  is a survival of motor neuron 2-directed RNA splicing modifier. In clinical trials, the most common adverse events included fever, diarrhea, rash, ulcers of the mouth area, joint pain (arthralgia) and urinary tract infections. Additional adverse events observed in the infantile-onset population included upper respiratory tract infection, pneumonia, constipation and vomiting.<unk>  was approved by the US Food and Drug Administration (FDA) in August 2020, for the treatment of adults and children two months of age or older. Developed in association with PTC Therapeutics and the SMA Foundation, it is marketed in the US by Genentech, a subsidiary of Roche.
Risdiplam
Lemborexant
Fenproporex
Tasimelteon
Fosaprepitant
Bocasan
Mitapivat
Pleconaril