None

None

None

None

None

Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

Primary Objectives: To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal function To assess the safety and tolerability of lademirsen (SAR339375) in subjects with Alport syndrome Secondary Objectives: To assess plasma pharmacokinetic (PK) parameters of the parent compound and its metabolites To assess the potential formation …

Cohort of Patients With Systemic Sclerosis Within the Framework of the RESO Reference Centre

Systemic sclerosis (SSc) is a rare form of connective tissue disease characterized by vascular involvement and the intensity of fibrosis. The lack of available treatment is largely due to the very fragmented understanding of the pathophysiology of SSc.

Rheumatology Patient Registry and Biorepository

To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.

The ATtune Knee Outcome Study

To accomodate dissatisfied patients with a total knee arthroplasty (TKA) and improve outcomes, several knee systems have been developed. The cemented ATTUNE TKA shows superiority over other established knee systems at short-term, abating with longer follow-up. There have been no studies reporting on the results of the uncemented version of …

RASopathy Biorepository

The RASopathies are a group of developmental disorders caused by genetic changes in the genes that compose the Ras/mitogen activated protein kinase (MAPK) pathway. New RASopathies are being diagnosed frequently. This pathway is essential in the regulation of the cell cycle and the determination of cell function. Thus, appropriate function …