Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.
Mallinckrodt plc announced that the company achieved target enrollment of 300 participants in the Phase 3 CONFIRM clinical study to evaluate the efficacy and safety of the investigational agent terlipressin in adult patients with Hepatorenal Syndrome Type 1 (HRS-1).
Pfizer announced plans to buy Therachon, a rare disease biotech company based in Basel, Switzerland.
Akcea Therapeutics Inc. and Ionis Pharmaceuticals Inc. announced that Waylivra received conditional marketing authorization from the European Commission as the only therapy for FCS.
Sanofi upheld a long-awaited return to growth with higher profits and revenues for first-quarter 2019, once again led by a stellar sales increase at the company’s rare diseases Genzyme unit.
Cambridge, Mass.-based Alnylam Pharmaceuticals indicated that the company’s ENVISION Phase III trial of givosiran was positive for acute hepatic porphyria (AHP).
Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.
The U.S. Food and Drug Administration approved ADMA Biologics’ Asceniv to treat primary humoral immunodeficiency disease (PIDD or PI) in adults and adolescents.
March 20, Horsham, PA — Dudnyk leaders announce the next phase in its evolution as an ever-growing organization by joining The Fishawack Group of Companies. Fishawack is one of […]
Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.
