by GlobeNewsWire | Aug 10, 2016 | Globe Newswire
Of the nine crossover subjects, eight responders demonstrated the maximum improvement measurable on the primary endpoint at one year 27 of 29, or 93%, of Phase 3 subjects that received voretigene neparvovec demonstrated a gain in functional vision at one year...
by GlobeNewsWire | Aug 9, 2016 | Globe Newswire
SAN RAFAEL, Calif., Aug. 09, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the pricing of an underwritten public offering of 7,500,000 shares of its common stock at a price to the public of $96.00 per share. The gross...
by GlobeNewsWire | Aug 9, 2016 | Globe Newswire
On track to enroll first patients for Phase I/II clinical trial of RGX-501 for the treatment of homozygous familial hypercholesterolemia in the second half of 2016Received FDA Rare Pediatric Disease Designation for RGX-121 for the treatment of Mucopolysaccharidosis...
by GlobeNewsWire | Aug 9, 2016 | Globe Newswire
CAMBRIDGE, Mass., Aug. 09, 2016 (GLOBE NEWSWIRE) — Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS), today announced dosing of the first...
by GlobeNewsWire | Aug 9, 2016 | Globe Newswire
Albany, NY, Aug. 09, 2016 (GLOBE NEWSWIRE) — GlobalDatas report “PharmaSphere: Emerging Biotechnologies Stem Cell Therapy”, provides strategic analysis of the global stem cells industry. It discusses key development trends, regulatory requirements in...