Rare Disease Research, LLC
Rare Disease Research, LLC
Atlanta, Georgia, United States8 results ยท Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-10-05 | NCT07486934 | |
| Randomized Study in Children and Adolescents With Migraine: Acute Treatment | Ear Diseases, Nausea, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Photophobia, Sensation Disorders, Signs and Symptoms, Signs and Symptoms, Digestive, brain disorder, central nervous system disorder, eye disorder, headache disorder, hearing disorder, hyperacusis, hypnic headache, migraine disorder, nervous system disorder, otorhinolaryngologic disease, vision disorder | Recruiting | Phase 3 | 2026-09-24 | NCT04649242 | |
| Long-term Extension of GTX-102 in Angelman Syndrome | 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 | 2026-09-09 | NCT06415344 | |
| Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Phase 4 | 2026-09-09 | NCT05232929 | |
| A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne 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 | Active, not recruiting | Phase 3 | 2026-08-28 | NCT05126758 | |
| Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | 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 1 / Phase 2 | 2026-08-11 | NCT05524883 | |
| A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Tourette syndrome, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, psychiatric disorder, tic disorder | Active, not recruiting | Phase 3 | 2026-07-22 | NCT06021522 | |
| A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Phase 3 | 2026-05-22 | NCT05067790 |