Huntington disease (HD) is a hereditary neurodegenerative condition characterized by progression of motor, cognitive, and psychiatric abnormalities. Despite the lack of definitive medications, current research efforts are actively assessing novel pharmaceutical interventions through phase III and IV clinical trials to mitigate the limited effectiveness of existing therapeutic approaches. The primary objective of these trials is to enhance symptom management and improve the overall quality of life for individuals diagnosed with HD. These trials show potential for development of further efficacious therapeutic interventions in future. To identify and provide details about medications tested in completed phase III and IV clinical trials for managing HD in adults. Publicly available and relevant phase III and IV trials registered at ClinicalTrials.gov analyzed. Usage of the trialed medications for HD reviewed. As of November 10, 2023, there were 242 phase III and IV clinical trials related to HD. Eight clinical trials from these met the inclusion criteria for the current study. The medications used in phase III and IV trials are minocycline, valbenazine, deutetrabenazine, tominersen, pridopidine (phase III), and memantine (phase IV). Evaluating phase III and IV clinical studies on HD highlights the importance of tailored approaches for each patient's unique disease presentation. Current medications aim to manage HD symptoms, potentially improving outcomes and reducing disease progression risks. The growing emphasis on specific approaches reflects a better understanding of HD's diverse symptoms, presenting opportunities for more effective and personalized treatment strategies.
Download full-text PDF |
Source |
---|---|
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11688028 | PMC |
http://dx.doi.org/10.1097/MD.0000000000041073 | DOI Listing |
J Am Coll Cardiol
March 2025
National Amyloidosis Centre, University College London, Royal Free Hospital, London, United Kingdom.
Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed chronic disease associated with progressive heart failure that results in impaired quality of life, repeated hospitalizations, and premature death. Acoramidis is a selective, oral transthyretin stabilizer recently approved by the U.S.
View Article and Find Full Text PDFJ Mol Model
March 2025
Departamento de Ciencias Químicas, Facultad de Ciencias Exactas, Universidad Andres Bello (UNAB), Av. República 275, Santiago, 8370146, Región Metropolitana, Chile.
Context: The conversion of carbon dioxide into methanoic acid through direct hydrogenation with H in the gas phase implies overcoming a high activation energy (more than 60 kcal mol ) that makes the process kinetically infeasible. In this study, the use of the [(PY Me )Mo(III)(H)(OH)] complex instead of H lowered the activation energy of the hydrogenation by 98.5%.
View Article and Find Full Text PDFCancer Biol Med
March 2025
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Objective: Our previous studies have indicated potentially higher proliferative activity of tumor cells in Chinese patients with mantle-cell lymphoma (MCL) than those in Western. Given the success and tolerability of R-DA-EDOCH immunochemotherapy in treating aggressive B-cell lymphomas, we designed a prospective, phase 3 trial to explore the efficacy and safety of alternating R-DA-EDOCH/R-DHAP induction therapy for young patients with newly diagnosed MCL. The primary endpoint was the complete remission rate (CRR) at the end of induction (EOI).
View Article and Find Full Text PDFCurr Opin Oncol
March 2025
Centre Léon Bérard, Lyon, France.
Purpose Of Review: Recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) remains a significant therapeutic challenge due to its biological complexity and clinical heterogeneity. This review addresses the relevance of exploring novel therapeutic approaches, particularly in light of recent advances and the persistent unmet needs in patient outcomes.
Recent Findings: Recent phase II and III clinical trials have highlighted promising strategies, including combinations of immunotherapy with targeted therapies, antibody-drug conjugates (ADCs), HPV vaccines, dual immunotherapy approaches, and therapies targeting the tumor microenvironment.
Indian J Otolaryngol Head Neck Surg
February 2025
Department of Otorhinolaryngology and Head and Neck Surgery, Father Muller Medical College, Mangalore, India.
Simulation, in medical education has provided a novel way to impart education which will help students acquire the required skills to achieve their learning objectives. The aim of our study was to evaluate the performance of students exposed to otoscopy simulator-based learning versus traditional learning with observation and also to know the students' perceptions via a questionnaire at the end of the module. This was a prospective interventional study, conducted on third-year medical students, sixty in number, divided into two groups by the lottery method.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!