Uncommon Cures

Chevy Chase, Maryland 20815

5 studies enrolling now · 8 studies all time

What they study most

Achondroplasia, Friedreich Ataxia, Homocystinuria, OTC Deficiency, OTCD, Ornithine Transcarbamylase Deficiency, PKU, Phenylketonuria, Prader-Willi Syndrome

Studies at this site

• A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment — Recruiting now
• A Study of TYRA-300 in Children With Achondroplasia: BEACH301 — Recruiting now
• An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia — Recruiting now
• A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD — Recruiting now
• A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia — Stopped early
• A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE) — Enrolling by invitation
• Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq — Running, not enrolling
• An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) — Withdrawn before enrolling