Found 7 clinical trials
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 …
- 0 views
- 19 Feb, 2024
- 23 locations
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.
- 0 views
- 19 Feb, 2024
Rheumatology Patient Registry and Biorepository
To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.
- 0 views
- 19 Feb, 2024
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 …
- 0 views
- 19 Feb, 2024
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 …
- 0 views
- 19 Feb, 2024
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome. During childhood, patients with rare genetic syndromes receive multidisciplinary and specialized medical care; they usually receive medical care from 3-4 medical specialists. …
- 0 views
- 19 Feb, 2024
MPA AUC Monitoring in Patients Receiving MMF for Diffuse Cutaneous or Pulmonary Involvement in Systemic Sclerosis
To define a target value of AUC MPA to improve the modified Rodnan score and / or respiratory impairment (DLCO or FVC) at one year in patients receiving MMF for the treatment of diffuse cutaneous or interstitial lung damage of systemic sclerosis.
- 0 views
- 19 Feb, 2024