This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical ...
The primary aim is to utilize near-infrared spectroscopy (NIRS) in patients under general anesthesia to measure changes in brain blood flow in the bilateral somatosensory cortices and the prefrontal ...
The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1, ...
Laparoscopic cholecystectomy is associated with considerable postoperative pain and surgeon-administered local anesthetic infiltration is the standard practice for achieving post-operative analgesia. ...
Although the last decade has brought major advances with respect to our knowledge of certain risks associated with fetal exposure to psychiatric medications, critical information regarding the long ...
There is currently no standard of care or best practice for managing post-operative pain for patients undergoing hip arthroscopy. Perioperative pain with these procedures can be substantial. It has ...
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated ...
This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lymphoblastic leukemia. Part 1 of the study will optimize ...
The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do ...
This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent subjects who have ...