The Fast Track designation underlines the need for treatment options to fulfill the unmet medical needs of people affected by primary sclerosing cholangitis.
LION-101 is a recombinant adeno-associated virus based vector being developed as a one-time intravenous infusion for Limb-Girdle Muscular Dystrophy Type 2I/R9.
Underscores potential for momelotinib to address unmet needs of patients with Intermediate/High-Risk myelofibrosis who have previously received a JAK inhibitor