Necker-Enfants Malades Hospital

Paris 75015

3 studies enrolling now · 6 studies all time

What they study most

Advanced Systemic Mastocytosis (AdvSM), Aggressive Systemic Mastocytosis (ASM), Angelman Syndrome, Arterial Calcification, Autosomal Recessive Hypophosphatemic Rickets Type 2, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Gene Mutations, Mast Cell Leukemia (MCL), Phosphomannomutase 2 Deficiency, Pseudoxanthoma Elasticum, SM With an Associated Hematologic Neoplasm (SM-AHN), Urinary Incontinence

Studies at this site

• A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) — Recruiting now
• (Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis — Recruiting now
• HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome — Recruiting now
• ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency — Recruiting now
• Natural History Study Protocol in PMM2-CDG (CDG-Ia) — Completed
• Study of OnabotulinumtoxinA (BOTOX®) for Urinary Incontinence Due to Neurogenic Detrusor Overactivity in Pediatric Patients — Completed