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A Retrospective and Prospective Natural History of Genetic Vasculopathies
This study will combine retrospective review of medical records from patients with ACTA2 and ongoing collection of clinical data using standardized instruments…
Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults: Exploratory Observational Analysis of Novel Robotic Control Strategies
People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and le…
Testing LiverWatch, a Home-Based Remote-Monitoring Intervention for Advanced Liver Disease
Remote healthcare monitoring for cirrhosis has shown promise in overcoming barriers to accessing specialty care, improving healthcare quality, and reducing mor…
The Utilization of a Shoe Insert on Individuals With Unilateral Greater Trochanteric Pain Syndrome on Single Leg Stance, Biomechanics, and Pain Intensity
We hypothesize that the addition of a unilateral neutral shoe insert combined with standard physical therapy (PT+SI) will have greater improvements in pain and…
Comparing the Effects of Pneumatic Compression and Blood Flow Restriction Therapy on Muscle Recovery
Both BFR and intermittent pneumatic compression are purported to decrease symptoms associated with exercise induced muscle damage (EIMD) that cause delayed ons…
Imaging Skeletal Muscle Mitochondrial OXPHOS Activity In Acute Lymphoblastic Leukemia Survivors
The participants are being asked to take part in this trial, because the participant is a survivor of childhood cancer or agreed to be part of a volunteer grou…
Temporo-masseteric Nerve Block in Post-wisdom Tooth Extraction Pain- a Randomized Study
The proposed design is a triple-blind, placebo-controlled randomized prospective phase II trial to assess the feasibility of employing the Temporo-masseteric N…
Phase 1/2 Study of Erdafitinib Intravesical Delivery System (TAR-210) in Participants With Non-Muscle-Invasive or Muscle-Invasive Bladder Cancer
The purpose of the study in Part 1 (dose escalation) and in Part 2 (dose expansion) is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) and evaluate pr…
Feasibility and Correlation of Functional Muscle Strength Tests and Objective Frailty Measures With Clinical Frailty Scale in Patients Undergoing Major Surgery
The goal of this observational study is to learn if simple tests for frailty and muscle strength can help predict which older adults (age 65 and older) are at …
Imaging Genetics of Laryngeal Dystonia
The contribution of genetic risk factors to the development of focal dystonias is evident. However, understanding of how variations in the causative gene expre…
A Phase 3, Randomized Study Evaluating the Efficacy and Safety of TAR-210 Erdafitinib Intravesical Delivery System Versus Single Agent Intravesical Chemotherapy in Participants With Intermediate-risk Non-muscle Invasive Bladder Cancer (IR-NMIBC) and Susceptible FGFR Alterations
The main purpose of this study is to compare the disease-free survival between participants receiving treatment with TAR-210 versus investigator's choice of in…
A Phase 1/2 Open-label Intrathecal Administration of MELPIDA to Determine Its Safety and Efficacy for Patients with Spastic Paraplegia Type 50 (SPG50) Caused by Mutation in the AP4M1 Gene.
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal inj…