Rare Disease Research

Atlanta, Georgia 303329

5 studies enrolling now · 30 studies all time

What they study most

ARG1 Deficiency, Angelman Syndrome, Atrophies, Facioscapulohumeral, Atrophy, Facioscapulohumeral, Becker Muscular Dystrophy, DMD, Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy (DMD), Dystrophies, Facioscapulohumeral Muscular, Dystrophies, Landouzy-Dejerine, Dystrophy, Facioscapulohumeral Muscular, Dystrophy, Landouzy-Dejerine

Studies at this site

• A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) — Recruiting now
• Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients — Recruiting now
• A Study of Pitolisant in Patients With Prader-Willi Syndrome — Recruiting now
• NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) — Recruiting now
• Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) — Running, not enrolling
• Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) — Running, not enrolling
• Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy — Enrolling by invitation
• Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping — Available (expanded access)
• CureDuchenne Link®: A Resource for Research — Completed
• AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) — Running, not enrolling
• A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy — Running, not enrolling
• First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria — Completed
• Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) — Completed
• Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) — Running, not enrolling
• AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD — Running, not enrolling
• An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM) — Running, not enrolling
• A Phase 2b Study of Zagociguat in Patients With MELAS — Completed
• A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy — Running, not enrolling
• A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS — Running, not enrolling
• Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 — Completed
• A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome — Completed
• Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome — Completed
• A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) — Running, not enrolling
• A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy — Completed
• Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 — Completed
• An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) — Withdrawn before enrolling
• Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) — Stopped early
• Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) — Stopped early
• A Study of RVT-1401 in Myasthenia Gravis (MG) Patients — Completed
• A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids — Stopped early