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A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome).

The objective of the study is to quantify endurance and respiratory function in subjects with MPS IV A and to better characterize the spectrum of symptoms and biochemical abnormalities in MPS IV A disease over time.

Cytokines, autoantibodies and astrocytes in the pathology of Aicardi-Goutieres syndrome (AGS)

The objectives of the complete NIMBL study are as follows:Objective 1. Gain insight into the natural history of AGS and RVCL* Create a registry of paediatric and adult AGS and RVCL patients, and develop a network of clinicians caring for these…