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Amylyx Update on Development of AMX0114, an Antisense Oligonucleotide Targeting Calpain-2
Lead Author: Lauren Kett, MD, PhD (Presenter)
Published: ALS ONE’s 7th Annual ALS Research Symposium
Date: November 14, 2024
Results from the HELIOS Trial: A Phase 2 Open-Label Study Evaluating an Oral, Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol in Wolfram Syndrome
Lead Author: Amy Viehoever, MD, PhD (Presenter)
Published: Child Neurology Society Annual Meeting
Date: November 11 - 14, 2024
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: Lauren Kett, MD, PhD (Presenter)
Published: Northeast ALS Consortium (NEALS) Annual Meeting
Date: October 23, 2024
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: Nathalie Erpelding, PhD (Presenter)
Published: 9th International Wolfram Syndrome Symposium
Date: October 23, 2024
Results from A Phase 2, Open-Label Study Evaluating an Oral, Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol (PB&TURSO) in Wolfram Syndrome (HELIOS)
Lead Author: Lahar Mehta, MD (Presenter)
Published: International Society for Pediatric and Adolescent Diabetes (ISPAD) 50th Annual Congress
Date: October 17, 2024
ORION: A Global Phase 2b/3, Randomized, Double-Blind, Placebo-Controlled Trial of AMX0035 in Progressive Supranuclear Palsy (A35-009)
Lead Author: Anne-Marie Wills, MD
Published: International Parkinson and Movement Disorder Society
Date: October 1, 2024
Biological Effects of Sodium Phenylbutyrate and Taurursodiol in Alzheimer's Disease
Lead Author: Steven E. Arnold
Published: Alzheimer’s & Dementia: Translational Research & Clinical Interventions, a journal of the Alzheimer’s Association
Date: August 9, 2024
Preclinical Development of AMX0114, an Antisense Oligonucleotide Targeting Calpain-2
Lead Author: Evan Mizerak (Presenter)
Published: Oligonucleotides for CNS Summit
Date: July 10, 2024
Learnings from the CENTAUR and PHOENIX Trials: Biomarker Results
Lead Author: Sabrina Paganoni, MD, PhD (Presenter)
Published: European Network to Cure ALS (ENCALS) Meeting 2024
Date: June 17 - 20, 2024
Results From the Global Phase 3 Trial Evaluating Sodium Phenylbutyrate and Ursodoxicoltaurine in ALS
Lead Author: Leonard H. van den Berg, MD, PhD, and Sabrina Paganoni, MD, PhD (Presenters)
Published: European Network to Cure ALS (ENCALS) Meeting 2024
Date: June 17 - 20, 2024