Children's Health Ireland (CHI) at Temple Street

Dublin D01 XD99

2 studies enrolling now · 3 studies all time

What they study most

Homocystinuria, Muscular Dystrophy, Duchenne

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 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
• A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) — Running, not enrolling