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Next Steps in Development for AMX0114: An Antisense Oligonucleotide Targeting Calpain-2, a Critical Effector of Axonal Degeneration

Lead Author: Joshua Cohen

Published: 2024 MDA Clinical & Scientific Conference (MDA)

Date: March 3 - 6, 2024

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A Joint Model for Assessing Mortality-Adjusted Progression (MAP) in Amyotrophic Lateral Sclerosis: Application to Clinical Trials of Sodium Phenylbutyrate and Taurursodiol

Lead Author: Ruben van Eijk

Published: International Symposium on ALS/MND (MNDA)

Date: December 6 - 8, 2023

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Effect of Sodium Phenylbutyrate and Taurursodiol on Plasma Concentrations of Neuroinflammatory Biomarkers in Amyotrophic Lateral Sclerosis: Results From the CENTAUR Trial

Lead Author: Robert Bowser, PhD & Jiyan An

Published: Journal of Neurology, Neurosurgery and Psychiatry

Date: December 2, 2023

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Rethinking Economic Evaluation in Amyotrophic Lateral Sclerosis Treatment: Embracing Alternative Model Conceptualizations

Lead Author: Sara Öhrman (Presenter)

Published: International Society for Pharmacoeconomics and Outcomes Research (ISPOR)

Date: November 12 - 15, 2023

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Proteomic Analyses in the 24-Week PEGASUS Trial Using the Olink Platform: Providing Insight Into the Biologic Activity of Sodium Phenylbutyrate and Taurursodiol in Alzheimer’s Disease

Lead Author: Lahar Mehta, MD (Presenter)

Published: Clinical Trials on Alzheimer’s Disease (CTAD)

Date: October 24 - 27, 2023

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Design of a Global Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial of AMX0035 in Progressive Supranuclear Palsy (A35-009 ORION)

Lead Author: Günter Höglinger, MD

Published: Neuro2023

Date: October 19 - 20, 2023

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Analysis of Sodium Phenylbutyrate and Taurursodiol Survival Effect in ALS Using External Controls

Lead Author: Sabrina Paganoni, MD, PhD & Melanie Quintana, PhD

Published: Annals of Clinical and Translational Neurology

Date: October 9, 2023

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An In Vitro Recovery Study of Sodium Phenylbutyrate and Taurursodiol From 3 Types of Dosing Containers and Various Percutaneous Endoscopic Gastrostomy Feeding Tubes

Lead Author: Machelle Manuel, PhD

Published: Northeast ALS Consortium Annual Meeting (NEALS)

Date: October 4 - 6, 2023

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Development of a Composite Diagnostic Biomarker for Amyotrophic Lateral Sclerosis: Experimental Approach and Progress to Date

Lead Author: Jamie Timmons, MD

Published: Northeast ALS Consortium Annual Meeting (NEALS)

Date: October 4 - 6, 2023

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Novel Neuroprotective Strategies in Human Neuron Models of ALS/FTD: Evaluating Antisense Oligonucleotide Therapies, including AMX0114

Lead Author: Sami Barmada, MD, PhD

Published: Northeast ALS Consortium Annual Meeting (NEALS)

Date: October 4 - 6, 2023

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