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Interventions with Music in Pectus Excavatum Treatment

This research will investigate a non-pharmacological, low-cost, durable and safe method during and after pectus excavatum surgery, to alleviate postoperative pain and thereby reduce the dose of analgesics used. Our secondary aim is to investigate…

A randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva

Primary Objective:The primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).The primary efficacy objective of the study is to assess…

A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

This study has been transitioned to CTIS with ID 2023-510353-42-00 check the CTIS register for the current data. The purpose of this first-in-human study is to evaluate the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics…

Inspiratory muscle training in patients with nemaline myopathy

The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients. The secondary objective is to determine respiratory muscle function in nemaline myopathy…