pharmaphorum45m
Pfizer drops Beqvez, leaving its gene therapy cupboard bare
Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a ...
pharmaphorum12m
FDA clears first therapy for rare disease CTX from Mirum
Mirum Pharma's Ctexli has become the first FDA-approved therapy for cerebrotendinous xanthomatosis (CTX), an ultra-rare form ...