Utrecht, Netherlands and Vienna, Austria – Neuroblastoma mainly affects toddlers and young children – in the EU region there are 1500 new cases per year. Neuroblastoma is a malignant tumor of the peripheral nervous system and around 50% of patients are high-risk cases. Recurrences occur frequently, and conventional therapies are...
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CAMBRIDGE, Mass. — NeuroBo Pharmaceuticals, Inc. (Nasdaq: NRBO), a clinical-stage biotechnology company focused on transforming cardiometabolic diseases, today announced the completion of enrollment of Part 1 of its two-part, Phase 2a clinical trial evaluating the efficacy and safety, of DA-1241, a novel G-Protein-Coupled Receptor 119 (GPR119) agonist for the treatment...
– Highly Statistically Significant Reduction in Chorea Movements (p < 0.0001) as Measured by the Unified Huntington’s Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) Score – Placebo-Adjusted Mean Reduction in TMC Score of 3.2 Units in Valbenazine-Treated Patients – Company Plans to Submit Supplemental New Drug Application to U.S....
SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the launch of WHAT THE C@H?!, an educational initiative that aims to close the gap in the need for helpful information about congenital adrenal hyperplasia (CAH) and acknowledges the frustrations and challenges experienced by the community in managing the condition. “We...
SAN DIEGO – Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced its support of Mental Health Month and commitment to raising awareness of and supporting people living with tardive dyskinesia (TD). Some people living with a mental health condition may also experience TD, an involuntary movement disorder that may develop after...
SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the U.S. Food and Drug Administration has approved INGREZZA® SPRINKLE (valbenazine) capsules, a new oral granules formulation of INGREZZA® (valbenazine) capsules prescribed for the treatment of adults with tardive dyskinesia and chorea associated with Huntington’s disease. INGREZZA SPRINKLE provides...
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...
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 initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome, which showed that NGN-401 was...
NEW YORK – Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced results of a well-controlled non-human primate study, which demonstrate that route of adeno-associated virus (AAV) administration plays a critical role in optimizing gene therapy delivery to key...