SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the publication of the first peer-reviewed expert recommendations to guide glucocorticoid dose reduction in patients with classic congenital adrenal hyperplasia treated with CRENESSITY® (crinecerfont). Published in The Journal of Clinical Endocrinology & Metabolism, the recommendations address a critical unmet need as real-world use of CRENESSITY expands in...
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SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the presentation of new two-year data from the Phase 3 CAHtalyst® Pediatric study demonstrating durable androgen control, sustained decreases in glucocorticoid (GC) doses and meaningful improvements in clinical outcomes associated with excess androgens and long-term GC exposure in children and adolescents with classic congenital...
SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced publication of the KINECT-PRO™ Phase 4 open-label study results demonstrating that treatment with once-daily INGREZZA® (valbenazine) capsules resulted in substantial and sustained improvements in patient-reported quality of life and functionality, alongside improvements in clinician-rated tardive dyskinesia (TD) movement severity. Improvements were observed regardless of underlying psychiatric diagnosis...
CHARLESTON, S.C. – Neuroene Therapeutics announced that the FDA has granted orphan drug designation for the company’s proprietary compound: NT102. NT102 was developed as part of a novel patented platform of mitochondrial and neuroprotective chemicals for the treatment of neurological diseases such as pharmacoresistant epilepsies. In preclinical studies funded by...
NEW YORK – Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) for patients...
Oral presentation at ASGCT Annual Meeting to describe algorithm to monitor, detect and treat HLH, which can be adopted for AAV gene therapy NEW YORK, NY – Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today...
NEW YORK, NY — Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to NGN-401, an investigational gene therapy in late-stage clinical...
NEW YORK, NY – Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the first participant was dosed in the Embolden™ registrational clinical trial of NGN-401, a gene therapy in development for the treatment...
NEW YORK, NY — Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today provided an update on recent achievements for NGN-401 gene therapy for the treatment of Rett syndrome and plans for multiple value-creating catalysts anticipated...
NEW YORK, NY — Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced details of Embolden™, the Company’s registrational clinical trial designed to evaluate NGN-401 gene therapy for the treatment of females with Rett syndrome...