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Hypertonic saline in primary ciliary dyskinesia: a pilot study

We hypothesize that hypertonic saline nebulizations improve respiratory symptoms and quality of life in PCD patients.

A Phase 4 Randomised, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Centre Study of Colesevelam as Add-on Therapy in Patients with Familial Hypercholesterolaemia.

This study is being conducted in patients with familial hypercholesterolaemia (FH) to:1. Assess the efficacy of colesevelam added to a maximal tolerated and stable regimen of statin and ezetimibe in further decreasing the low-density lipoprotein (…

Treating Leg Symptoms in Women with X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance

This study has been transitioned to CTIS with ID 2024-518989-27-00 check the CTIS register for the current data. Objective: Primary Aim (PHASE 1):To determine the prevalence of RLS in women with ALD.Secondary Aim (PHASE 2):To determine whether in a…

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Praluent on Neurocognitive Function in Patients with Heterozygous Familial Hypercholesterolemia or with Non-Familial Hypercholesterolemia at High and Very High Cardiovascular Risk

The primary objective of the study is to evaluate neurocognitive function with use of Praluent after 96 weeks of treatment versus placebo.