Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHDFSHD133 moreA Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy...
Spinal Muscular AtrophyThe study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials...
Spinal Muscular Atrophy (SMA)This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 15 years from the date of OAV101 administration in the parent study.
Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual...
Neuromuscular DiseaseDuchenne Muscular Dystrophy3 moreOur study is a randomized controlled study and the subjects included in the study will be divided into three groups as virtual reality training, biofeedback training, and conventional rehabilitation.
Volume Support/Assist Control Mode Ventilation and Diaphragmatic Atrophy
Mechanical Ventilation ComplicationThe objective of the study is to determine how controlled mode ventilation and support mode ventilation impact ventilator-free days and diaphragmatic atrophy.
Prevention of Recurrent UTI Using Vaginal Testosterone Versus Placebo Placebo
Recurrent Urinary Tract InfectionVaginal Atrophy1 moreWomen over the age of 60 years have an estimated 10 to 15 % risk of recurrent urinary tract infections (UTI). This is believed to be due to hormonally induced changes in the vaginal flora associated with menopause. After menopause, there is a chemical changes in the vagina that may predispose to bacterial infections. The role of vaginal estrogen creams to restore vaginal atrophy and prevent urinary tract infections has been well characterized. Vaginal testosterone (VT) application use in postmenopausal breast cancer patients on aromatase inhibitors have been shown to improve vaginal pH, vaginal atrophy symptom scores, dyspareunia, and vaginal dryness. Although testosterone has been used to improve sexual function in postmenopausal women, the effects of VT on vaginal flora and recurrent UTIs are unknown. The purpose of this study is to determine whether topically applied vaginal testosterone cream is more effective than placebo in reducing the incidence of urinary tract infections in postmenopausal women with recurrent urinary tract infections and to ascertain the effects of topical estrogen on the vaginal pH and flora.
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults...
Multiple System AtrophyThe primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA). The secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).
Laser Vaginal Treatment for GSM
Menopausal Urethral AtrophyVulvar AtrophyGenitourinary Syndrome of Menopause (GSM) due to low estrogen levels affects about half of post-menopausal women and may have a dramatic impact on women's quality of life. Women complain of vaginal dryness, itching, discomfort, malodour, painful intercourse and may have urinary urgency, irritation, bladder/urethral pain and recurring bladder infections. First-line therapies include vaginal moisturizers, lubricants and estrogen (either oral or with vaginal cream/tablets). While these therapies are effective, the ongoing costs and the resistance to the indefinite use of vaginal creams/inserts is a challenge to the continued use of these therapies. Recently, an innovative laser therapy has been used to treat women with GSM. A randomized controlled trial (RCT) to study how effective the laser is to treat women with GSM is planned.
A Multiple Dose Study of AVD-104 for Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration...
Geographic Atrophy of the MaculaMacular DegenerationInvestigate the Safety, Pharmacokinetics, and Treatment effects of Single and Multi-dose of Intravitreal AVD-104 in participants with geographic atrophy secondary to age-related macular degeneration.
Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy
Geographic AtrophyThis is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD). This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 63 subjects.