AXLRx Sanofi intelligence — Rare Disease in United States: 3 reports covering Rare Disease in United States.
Two next-generation ERTs, avalglucosidase alfa and cipaglucosidase alfa plus miglustat, move to displace alglucosidase alfa across the US late-onset Pompe market.
Five FDA-approved Gaucher type 1 therapies (three IV enzyme replacement vs two oral substrate reduction) and eliglustat's oral first-line pivot.
Two IV enzyme replacement therapies meet an oral chaperone that only ~35–50% of patients can take — and pegunigalsidase now challenges Fabrazyme's two-decade lead.