National Neuromuscular Research Institute | Austin, TX
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Active trials
The primary purpose of this study is to measure the efficacy and safety of efgartigimod intravenously (IV) compared to placebo in participants with A...
The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular...
This study is researching an experimental combination treatment with pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on pa...
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque...
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...
This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs placebo administered twice a day (BID) for 21 days in ambulat...
This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.
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