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European Spinocerebellar Ataxia Type 3/Machado*Joseph Disease Initiative

To set up a trial*ready cohort by bringing together 7 European cohorts and 1 US cohortand include new subjects, which together comprise more than 800 subjects with the developmentand validation of innovative assessment instruments and disease…

A randomized, partially-blinded, active-controlled multicenter study of secukinumab to demonstrate reduction of radiographic progression versus GP2017 (adalimumab biosimilar) at 104 weeks and to assess the long term safety, tolerability and efficacy up to 2 years in patients with active ankylosing spondylitis

The purpose of this study is to demonstrate the impact on progression of structural damage in the spine as measured by the mSASSS in patients with AS. Data from this study will be used to support the submission of an AS label extension to include a…

*The Experience Sampling Method (ESM):
a real-time Patient-Reported Outcome Measure for symptom assessment in patients with overactive bladder syndrome.*

This project will be subdivided into three sections:Part I. Comparison of the ESM vs. SR-BDs and other retrospective questionnaires in the assessment of urological complaints in OAB such as urgency. Part II. Validation of new patient-reported…

A SYSTEMS BIOLOGY APPROACH TO OCULAR INFLAMMATION

Systems biology is an interdisciplinary approach that systematically describes the complex interactions between all the parts in a biological system, with a view to elucidating new biological rules capable of predicting the behavior of the…

A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (formerly ALXN1101) in Pediatric Patients with Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated with Recombinant Escherichia coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)

Primary:• To evaluate the safety of ORGN001 (formerly ALXN1101) over the first 6 months of treatmentSecondary:• To characterize the pharmacokinetics (PK) of increasing doses of ORGN001 (formerly ALXN1101) • To evaluate the effect of ORGN001 (…

Evaluation of the Neurologic Assessment in Neuro-Oncology (NANO) criteria among brain cancer patients

The development of an objective, standardized and validated tool for the assessment of clinical outcome of brain tumor patients