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Ultragenyx_GTX-102-CL301

GTX-102 is a human ubiquitin-protein ligase E3A antisense (UBE3A-AS) transcript-targeted antisense oligonucleotide (ASO) intended for the treatment of Angelman syndrome (AS). Findings from the Phase 1/2 dose-finding study (GTX-102-001) suggest a positive efficacy signal and ...


Other

Nellcor Vs. Masimo Pulse Oximetry Performance Comparison in Newborns

The purpose of this study is to compare heart rate and oxygen saturation performance between 2 pulse oximeters (Nellcor and Masimo, both approved by the U.S. Food and Drug Administration) in newborns. Pulse oximeters are ...


Other

DMX-200 in Patients With Focal Segmental Glomerulosclerosis

This study will evaluate an experimental medication called DMX 200 (repagermanium) for people with focal segmental glomerulosclerosis (FSGS). FSGS is a progressive kidney disease in which the filters (glomeruli) of the kidneys become inflamed and ...


Kidney

COG, AREN2231, Ph3, Open, Wilms Tumors

1.1 Primary Aims 1.1.1 Stage I Nephrectomy Only Stratum 2. To maintain EFS for Stage I FHWT patients without adverse biology who are also (1) 2 to < 4 years of age, OR (2) age ...