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147 Results
Completed
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
Rett Syndrome is a developmental disorder primarily if not exclusively affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of …
Baylor Role: Collaborator
Active, Not Recruiting
Phenylbutyrate Therapy for Maple Syrup Urine Disease
Maple syrup urine disease is a severe inborn error of amino acid metabolism caused by deficiency of the mitochondrial branched-chain alpha-ketoacid dehydrogenase complex (BCKDC) resulting in the accumulation of branched-chain …
Completed
Adherence in Pediatric Multiple Sclerosis
Disease modifying therapies (DMT) are widely used for children and adolescents with MS. Nonetheless, many pediatric patients continue to relapse and therefore require changes in therapy. We designed this research …
Baylor Role: Collaborator
Completed
Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)
This is an interventional, case control study to investigate the production of nitric oxide in patients with citrullinemia type I. Through the infusion of isotopes [15N2-ureido] arginine, [5-13C,4, 4, 5, …
Baylor Role: Lead Sponsor
Withdrawn
Umbilical Cord Blood in the Treatment of Stroke in Children.
This study will enroll ten pediatric patients who have their umbilical cord blood banked with Cord Blood Registry, Inc. (CBR), who have been diagnosed with an arterial ischemic stroke (AIS), …
Baylor Role: Collaborator
Active, Not Recruiting
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Dyskeratosis congenita (DC) is an inherited multisystem disorder, which classically presents with a clinical triad of skin pigment abnormalities, nail dystrophy, and oral leukoplakia. DC is part of a spectrum …
Baylor Role: Collaborator
Completed
Neural and Behavioral Sequelae of Blast-Related Traumatic Brain Injury
Traumatic brain injuries (TBI) are a common occurrence from roadside blasts of improvised explosive devices (IEDs). Like civilian TBI, blast-related TBI can result from mechanical forces in which objects in …
Baylor Role: Lead Sponsor
Completed
Adaptive Portable Essential Tremor Monitor
The purpose of this study is to evaluate the utility of a portable motion sensor-based system designed to monitor essential tremor (ET) to better prescribe therapy to minimize symptoms and …
Baylor Role: Collaborator
Completed
Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
Subjects with AS, RTT or PWS and normal siblings (controls) will be recruited for study participation. Subjects will be recruited from the Rare Disease Clinical Research Network (RDCRN) consortium registries …
Baylor Role: Lead Sponsor
Completed
MER Versus MRI Guidance DBS in Parkinson's Disease
Patients who have been randomized into Group 1 will undergo DBS implantation while awake. On the morning of surgery, the patient will undergo placement of the stereotactic head frame under …
Baylor Role: Lead Sponsor