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An Observational, Longitudinal, Prospective, Long-Term Registry of Patients with hypophosphatasia ClinicalTrials.gov ID NCT02306720 Sponsor Alexion Pharmaceuticals, Inc. Information provided by Alexion Pharmaceuticals, Inc. (Responsible Party) Last Update Posted 2024-09-19 Study Overview In this...

A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment ofHyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA)in Adult and Pediatric Patient Populations ClinicalTrials.gov ID NCT05040178 Sponsor Recordati Rare Diseases Information provided...

A Phase 1, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants with Phenylketonuria ClinicalTrials.gov ID NCT05781399 Sponsor Jnana Therapeutics Information provided by Jnana Therapeutics (Responsible Party) Last Update...