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This trial is an extension of the antecedent trial ARGX-117-2002. It is a multicenter trial that has been designed to evaluate the long-term safety a...
This is a phase 2, randomized, double-blinded, placebo-controlled, parallel-group, multicenter trial to evaluate the safety and efficacy of 2 dose re...
The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutat...
The purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with IIM who previously p...
A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Sub...
This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammator...
This study is researching an experimental combination treatment with pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on pa...
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
The main purpose of this study is to evaluate the safety and efficacy of nipocalimab compared to placebo in delaying relapse in adults with chronic i...
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON s...
The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS
The purpose of this study is to evaluate the efficacy and safety of orally administered M5049 in idiopathic inflammatory myopathies, specifically der...
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose \[MAD\]) will be conducted to evaluate the safety and tolerability of ves...
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