Rare Disease Research, LLC.

Atlanta, Georgia 30329

4 studies enrolling now · 37 studies all time

What they study most

Acute Treatment of Migraine, Allan-Herndon-Dudley Syndrome, Angelman Syndrome, DM1, Dravet Syndrome, Dravet Syndrome (DS), Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy (DMD), Epilepsy, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Lennox-Gastaut Syndrome

Studies at this site

• Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 — Recruiting now
• Long-term Safety Study of Rimegepant in Pediatric Subjects for the Acute Treatment of Migraine — Recruiting now
• Randomized Study in Children and Adolescents With Migraine: Acute Treatment — Recruiting now
• Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease — Recruiting now
• Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) — Recruiting now
• Long-term Extension of GTX-102 in Angelman Syndrome — Enrolling by invitation
• A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder — Running, not enrolling
• Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — Running, not enrolling
• A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy — Completed
• Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults — Completed
• A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy — Running, not enrolling
• 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) — Running, not enrolling
• Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency) — Completed
• An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy — Stopped early
• A Study of Soticlestat in Adults and Children With Rare Epilepsies — Stopped early
• Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) — Completed
• A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) — Running, not enrolling
• Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) — Completed
• Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping — Running, not enrolling
• Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment — Stopped early
• An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy — Stopped early
• A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) — Completed
• Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) — Stopped early
• Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension — Completed
• Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System — Stopped early
• Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents — Completed
• A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) — Completed
• A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) — Stopped early
• Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy — Completed
• A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension — Stopped early
• Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy — Stopped early
• A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy — Stopped early
• A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies — Completed
• Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy — Completed
• An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy — Stopped early
• An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — APPROVED_FOR_MARKETING
• Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy — Completed