Ionis Gets FDA Approval for Rare Disease Drug Tryngolza
The FDA's nod to IONS' Tryngolza makes it the first approved treatment for FCS. It is also the company's first wholly-owned ...
SMA18h
DMTs have transformed SMA type 1 in Italy, nationwide study finds
Nearly 4 of every 5 infants in Italy born with SMA type 1 since the approval of disease-modifying therapies (DMTs) are alive, ...