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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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, California — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the presentation of the first expert consensus recommendations focused on screening, diagnosis and treatment of tardive dyskinesia among older adults in long-term care settings. Developed through a multidisciplinary Delphi panel, the recommendations address persistent gaps in recognizing and managing tardive dyskinesia in this higher-risk...
Most Studies Meeting the Literature Review Criteria Found That a Higher Glucocorticoid Dose was Statistically Significantly Associated with Adverse Clinical Outcomes Findings Presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research Annual Meeting SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) presented today a systematic literature review illustrating the...
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...
Indianapolis, Ind. – Alois Alzheimer recognized more than a century ago the role of neuroinflammation in the development of the disease that would bear his name. “If you actually go back to his original drawings, he was drawing the cells, which today we know are microglia,” immune cells of the...
WUHAN, China and SAN DIEGO, Calif. — Neurophth Therapeutics, Inc. announced today that the last patient has been enrolled in Phase I/II clinical trial of Opvika® (Esonadogene Imvoparvovec) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON). Neurophth is conducting a Phase I/II, single-arm, multi-center study aimed...
Jerusalem, Israel – In a compelling Genomic Press Interview published in Brain Medicine on September 25, 2024, Professor Hermona Soreq of the Hebrew University of Jerusalem in Israel unveils the profound implications of her groundbreaking research on the cholinergic system and small RNA regulators in brain-body communication. Prof. Soreq, holder...
Toronto, Canada – Researchers at The Hospital for Sick Children (SickKids) have found that inflammation in an immune cell may be responsible in part for some severe symptoms in a group of rare genetic conditions called lysosomal storage diseases (LSDs). LSDs affect about one in 7,700 live births worldwide. Children...
TORONTO, Canada – A new round of clinical trials supported by the Ontario Institute for Cancer Research (OICR) will harness a unique opportunity to test some of the newest treatment strategies for a range of different cancers. OICR has announced two new clinical trials as part of its Window-of-Opportunity (WOO)...