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An Open-label Single Center, Single Participant Study Of An Experimental Antisense Oligonucleotide Treatment For Amyotrophic Lateral Sclerosis (ALS) Due To CHCHD10 Gene Mutation
Rochester, MN
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10
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Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study To Evaluate Safety, Tolerability, PK And PD Of Antisense Oligonucleotide AMX0114 Administered To Adult Participants With Amyotrophic Lateral Sclerosis
Jacksonville, FL
Rochester, MN
This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).
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PLS Natural History Study (PLS)
Jacksonville, FL
Rochester, MN
The purpose of this study is to develop a natural history dataset and biorepository of early Primary Lateral Sclerosis (PLS) and well-established PLS cases for future clinical trials.
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