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Safety and Efficacy of Deutetrabenazine at High versus Lower Daily Dosages in the ARC-HD Study to Treat Chorea in Huntington Disease.
CNS Drugs
February 2025
Innovative Medicines and Global Clinical Development, Teva Branded Pharmaceutical Products R&D, Inc., West Chester, PA, USA.
Background: Huntington disease (HD) is a progressive neurodegenerative disease that causes psychiatric and neurological symptoms, including involuntary and irregular muscle movements (chorea). Chorea can disrupt activities of daily living, pose safety issues, and may lead to social withdrawal. The vesicular monoamine transporter 2 inhibitors tetrabenazine, deutetrabenazine, and valbenazine are approved treatments that can reduce chorea.
View Article and Find Full Text PDFJean-Marin Charcot died unexpectedly on August 16, 1893, at the age of 67, while on a journey to the Morvan for a short holiday with colleagues and friends. This article reports in detail circumstances of Charcot's journey, and his untimely death in a small and modest room in a secluded hotel in the French countryside. In Part 1, I describe the reasons for Charcot's choice to go on the journey, with an emphasis on his role in the recent and controversial "Panama Affair.
View Article and Find Full Text PDFA novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.
Haemophilia
November 2024
Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
Introduction: Despite the progress in gene editing platforms like CRISPR/Cas9 with the potential to transform the standard of care for haemophilia, the language used to explain and discuss gene editing is not aligned across the haemophilia community. Here, we present the objective and rationale for developing a clear, consistent, and globally aligned gene editing lexicon to address these communication gaps.
Methods: Effectively communicating complex gene editing concepts requires a clear and consistent vocabulary.
Methods for pragmatic randomized clinical trials of pain therapies: IMMPACT statement.
Pain
October 2024
Department of Clinical and Biomedical Sciences, Faculty of Health and Life Sciences, University of Exeter, Exeter, United Kingdom.
Pragmatic, randomized, controlled trials hold the potential to directly inform clinical decision making and health policy regarding the treatment of people experiencing pain. Pragmatic trials are designed to replicate or are embedded within routine clinical care and are increasingly valued to bridge the gap between trial research and clinical practice, especially in multidimensional conditions, such as pain and in nonpharmacological intervention research. To maximize the potential of pragmatic trials in pain research, the careful consideration of each methodological decision is required.
View Article and Find Full Text PDFExemestane plus everolimus and palbociclib in metastatic breast cancer: clinical response and genomic/transcriptomic determinants of resistance in a phase I/II trial.
Nat Commun
March 2024
Cancer Program, Eli and Edythe L. Broad Institute of MIT and Harvard, Cambridge, MA, USA.
The landscape of cyclin-dependent kinase 4/6 inhibitor (CDK4/6i) resistance is still being elucidated and the optimal subsequent therapy to overcome resistance remains uncertain. Here we present the final results of a phase Ib/IIa, open-label trial (NCT02871791) of exemestane plus everolimus and palbociclib for CDK4/6i-resistant metastatic breast cancer. The primary objective of phase Ib was to evaluate safety and tolerability and determine the maximum tolerated dose/recommended phase II dose (100 mg palbociclib, 5 mg everolimus, 25 mg exemestane).
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