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A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene

An Observational Study to Assess Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

Does Addition of Oscillatory Positive Expiratory Pressure (OPEP) Device to a Chest Physiotherapy Program Provide Further Health Benefits in Children With Bronchiectasis?

Effects of Manipulating Swimming Speed and Frequency on Swimming Economy in High-level Swimmers and Para-swimmers

The Comparative Efficacy and Safety of Sugammadex and Neostigmine in Reversing Neuromuscular Blockade After General Anesthesia: A Prospective Randomized Control Study

Role and Mechanism of Bifidobacterium Triple Viable Capsules in Improving Motor Symptoms in Patients With Mild to Moderate Parkinson's Disease: a Multicenter Randomized Clinical Study

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease

Comorbid Insomnia and Sleep Disordered Breathing in Patients Undergoing Cardiac Rehabilitation: Prevalence and Impact on Cardiovascular Risk Profile

COMT Inhibition as a Potential Therapeutic Target Among Individuals With Comorbid Alcohol Use Disorder and Attention-Deficit/Hyperactivity Disorder

D-cycloserine Augmented Treatment: Enhancing Extinction Learning in Youth With Tic Disorders

Efficacy of Suvorexant in Patients With Effectively Treated Restless Legs Syndrome and Persistent Chronic Insomnia: A Randomized Placebo-Controlled Crossover Trial

Phase II Study to Examine Precision Radiation in Patients With Pathogenic Mutations in ATM

Showing 1-12 of 521