Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades

Paris 75015

3 studies enrolling now · 3 studies all time

What they study most

Homocystinuria, Phenylketonuria

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 Sepiapterin in Participants With Phenylketonuria (PKU) — 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