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Background
Smith-Magenis Syndrome (SMS) is a genetic syndrome caused by mutations in the RAI1 gene or deletions of the 17p11.2 chromosome.…
An Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants with…
Introduction:
The melanocortin-4 receptor (MC4R) pathway is the principal regulator of mammalian energy balance and body weight. Originating in the…
RM-493-034: A 2-Stage (Open-Label Run-In Followed by Randomized Withdrawal), Double-Blind, Placebo-Controlled, Phase 2 Study of Setmelanotide In…
Long Term Extension Trial Of Setmelanotide (RM-493) For Patients Who Have Completed A Trial Of Setmelanotide For The Treatment Of Obesity Associated…
The goal of this study is to establish a database of pediatric patients with early-onset obesity who have been phenotypically well-characterized…
Recruitment for a study to learn about the experiences and perspectives of children with dystonia and their caregivers to learn what you think about…
A study on natural history and molecular mechanisms of neurodevelopmental and neuropsychiatric disorders, including EBF3-related Hypotonia, Ataxia,…
