Rare Disease Research
Rare Disease Research
Atlanta, Georgia, United States12 results · Page 1 of 1
Clinical trials
Swipe the table sideways for more columns.
| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy | Becker muscular dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Enrolling by invitation | Phase 2 | 2026-09-17 | NCT06066580 | |
| NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 2 | 2026-09-15 | NCT05996003 | |
| A Study of Pitolisant in Patients With Prader-Willi Syndrome | Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disorders of Excessive Somnolence, Dyssomnias, Imprinting Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Prader-Willi syndrome, Sleep Disorders, Intrinsic, chromosomal disorder, hereditary disease, intellectual disability, nervous system disorder, nutritional disorder, obesity disorder, overnutrition, psychiatric disorder, sleep-wake disorder | Recruiting | Phase 3 | 2026-08-25 | NCT06366464 | |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 3 | 2026-08-06 | NCT06617429 | |
| AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 2 / Phase 3 | 2026-07-21 | NCT05693142 | |
| Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, facioscapulohumeral muscular dystrophy 1, facioscapulohumeral muscular dystrophy 2, frontometaphyseal dysplasia 2, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, progressive muscular dystrophy | Active, not recruiting | Phase 2 | 2026-07-14 | NCT06547216 | |
| A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Phase 1 / Phase 2 | 2026-07-08 | NCT06138639 | |
| An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM) | Acid-Base Imbalance, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, MELAS syndrome, Mitochondrial encephalopathy, Nutritional and Metabolic Diseases, acidosis disorder, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, lactic acidosis, metabolic disease, mitochondrial encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, vascular disorder | Active, not recruiting | Phase 2 | 2026-07-07 | NCT06961344 | |
| AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Not stated | 2026-07-07 | NCT05683379 | |
| A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Phase 3 | 2026-06-01 | NCT05337553 | |
| A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS | Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Prader-Willi syndrome, chromosomal disorder, hereditary disease, intellectual disability, nervous system disorder, nutritional disorder, obesity disorder, overnutrition | Active, not recruiting | Phase 3 | 2026-04-06 | NCT06144645 | |
| A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 2 | 2025-11-12 | NCT05540860 |