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Phase 1
A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is saf…
Myotonic Dystrophy 1
ARTHEx Biotech S.L.NCT06300307
Myotonic Dystrophy Family Registry
The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in…
Myotonic DystrophyCongenital Myotonic DystrophyMyotonic Dystrophy 1+13 more
Myotonic Dystrophy FoundationNCT02398786
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history…
Myotonic Dystrophy 1DM1
Virginia Commonwealth UniversityNCT03981575