Tumor Pathology in Patients with Huntington’s Disease in a Specialized Center of Buenos Aires, Argentina (Preliminary Data)
Objective: To analyze the prevalence and type of neoplasms in patients with HD and to contrast it with the data of the general population in…Translating Preclinical Data to a Human Equivalent Dose for AMT-130 AAV Gene Therapy for Early Manifest Huntington’s Disease
Objective: To extrapolate the human equivalent dose (HED) for a Phase I/II clinical trial in adults to explore the safety, tolerability, and efficacy of striatally-administered…MRI, Clinical, and Neuropathological Findings after Bilateral Intra-striatal Administration of rAAV5-miHTT in Non-human Primates
Objective: To assess the effects of intra-striatal administration of a huntingtin protein (HTT) lowering gene therapy in macaca fascicularis non-human primates (NHP). Background: The aggregation…The Roche HD Natural History Study – an external comparator by design
Objective: The Roche Natural History (NH) study in Huntington’s disease (HD) (NCT03664804) is a prospective, longitudinal cohort study designed to provide further clinical validation of…Exploring the effects of intrastriatal AAV5-miHTT therapy on MRS signal and mutant huntingtin levels in the Q175FDN mouse model of HD
Objective: To assess the effects of intrastriatal AAV5-miHTT therapy on MRS signal and mutant huntingtin levels in the Q175FDN mouse model of HD. Background: Huntingtin…Huntington’s disease: A case review of Sub-Saharan Africans with diverse origins
Objective: To characterise the Huntingtons disease phenotype in Sub-saharan Africans. Background: Huntington’s disease (HD) is a debilitating neurodegenerative illness that leads to diminished cognitive function,…The History of Huntington Disease Description in Russia
Objective: The report provides detailed material on the history of the description of Huntington's disease (HD) in Russia. The first, unknown to a wide audience,…Sydenham’s Chorea: Realities and Challenges in Sub-Saharan Africa
Objective: The aim of this study was to describe the epidemiological, clinical, paraclinical and evolutive aspects of Sydenham's chorea. Background: Sydenham's chorea is a rare…Pallidotomy for Intractable Hemichorea in Sudden Hyperglicemic Pituitary Adenoma Patient
Objective: To prove that pallidotomy can be an alternative therapy for intractable chorea. Background: A 61 year old woman come with complaint sudden irregular movement…A randomized, double-blind, placebo-controlled Phase II Efficacy and Safety Study of the PDE10A inhibitor PF-02545920 in Huntington Disease (AMARYLLIS)
Objective: To assess efficacy on motor function, safety and tolerability of the phosphodiesterase-10A inhibitor PF-02545920 in Huntington disease (HD). Conclusion: PF-02545920 was generally safe and…
Abstracts from the International Congress of Parkinson’s and Movement Disorders.