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AMX0035 in Wolfram Syndrome: Program Progress and Emerging Insights
Lead Author: Jamie Timmons, MD (Presenter)
Published: Wolfram Syndrome UK’s International Researcher’s Symposium
Date: June 4, 2026
Phase 2 trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome
Lead Author: Fumihiko Urano, MD, PhD
Published: The Journal of Clinical Investigation
Date: May 15, 2026
Initial Safety Findings from the Phase 1 LUMINA Trial of AMX0114 in Amyotrophic Lateral Sclerosis
Lead Author: Lauren Kett, MD, PhD (Presenter)
Published: Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2026
Date: March 11, 2026
Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and Its Application in the Phase 1 LUMINA Trial
Lead Author: Robert Bowser, PhD (Presenter)
Published: 36th International Symposium on ALS/MND (MNDA)
Date: December 6, 2025
A Phase 1, Multicenter, Randomized, Placebo-Controlled Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of AMX0114 in Amyotrophic Lateral Sclerosis (LUMINA)
Lead Author: Sabrina Paganoni, MD, PhD (Presenter)
Published: 36th International Symposium on ALS/MND (MNDA)
Date: December 5, 2025
48-Week Results from the Phase 2, Open-Label Study Evaluating an Oral, Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol (PB&TURSO) in Wolfram Syndrome
Lead Author: Jamie Timmons, MD
Published: The Wolfram Syndrome UK and Snow Foundation Virtual Global Research and Trial Update
Date: November 15, 2025
LUMINA Study Update
Lead Author: Lauren Kett, MD, PhD and Sabrina Paganoni, MD, PhD (Co-Presenters)
Published: 2025 Annual NEALS Meeting
Date: October 8, 2025
Poster: Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS
Lead Author: Robert Bowser, PhD (Presenter)
Published: 2025 Annual NEALS Meeting
Date: October 8, 2025
Presentation: Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS
Lead Author: Robert Bowser, PhD (Presenter)
Published: 2025 Annual NEALS Meeting
Date: October 8, 2025
Update on the Phase 2b Portion of the ORION Trial in Progressive Supranuclear Palsy (PSP)
Lead Author: Anne-Marie Wills, MD (Presenter)
Published: International Congress of Parkinson’s Disease and Movement Disorders 2025
Date: October 7, 2025