SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia in ongoing clinical trials and real-world studies. The latest findings reinforce the importance of early treatment and the well-known, established efficacy and safety of VOXZOGO. The data will...

NOVATO, Calif. — Today Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301-treated patients (n=18) demonstrated a statistically...

EVRY, France — Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular dystrophy in patients treated at therapeutic doses in the first phase of an international multicenter clinical trial...