Harmony: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients with Myotonic Dystrophy Type 1, Followed by an Open-label Extension.
To evaluate the safety and efficacy of pitolisant compared with placebo in treating EDS in patients with DM1
Ages 18-65 with genetic confirmation of myotonic dystrophy type 1
Dr. Georgios Manousakis: Serum uric acid as biomarker of type I myotonic dystrophy
This is a pilot study aimed to determine whether there is any potential role of uric acid (UA) in the pathophysiology of myotonic dystrophy type 1 (DM1)
Ages 18-85 with genetic confirmation of myotonic dystrophy type 1