Oral masitinib doubles the life in patients with amyotrophic lateral sclerosis.
Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.
Olorophim by F2G and Shionogi & Co. is focused on invasive aspergillosis treatment.
Teprotumumab by Horizon Therapeutics can really help with proptosis and diplopia in Graves’ disease and beyond.
Novartis and its alpelisib will help with very rare diseases with malformations and excessive tissue growth.