Urea Cycle Disorders (UCD)Symptoms, Doctors, Treatments, Advances & More
Learn About Urea Cycle Disorders (UCD)
Common conditions include: Arginase Deficiency, Argininosuccinic Aciduria, Carbamoyl Phosphate Synthetase 1 Deficiency, Citrullinemia
Mureo Kasahara is a Transplant Surgeon practicing medicine in Tokyo, Japan. Kasahara is rated as an Elite expert by MediFind in the treatment of Urea Cycle Disorders (UCD). They are also highly rated in 11 other conditions, according to our data. Their clinical expertise encompasses Biliary Atresia, Urea Cycle Disorders (UCD), Liver Failure, Liver Transplant, and Hepatectomy.
Johannes Haberle practices practicing medicine in Zurich, Switzerland. Mr. Haberle is rated as an Elite expert by MediFind in the treatment of Urea Cycle Disorders (UCD). He is also highly rated in 18 other conditions, according to our data. His clinical expertise encompasses Urea Cycle Disorders (UCD), Ornithine Transcarbamylase Deficiency, Citrullinemia, Carbamoyl Phosphate Synthetase 1 Deficiency, and Liver Transplant.
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Gerald Lipshutz is a General Surgeon practicing medicine in Los Angeles, California. Dr. Lipshutz is rated as an Elite provider by MediFind in the treatment of Urea Cycle Disorders (UCD). He is also highly rated in 3 other conditions, according to our data. His clinical expertise encompasses Urea Cycle Disorders (UCD), X-Linked Creatine Deficiency, End-Stage Renal Disease (ESRD), Kidney Transplant, and Simultaneous Pancreas Kidney Transplant.
Summary: This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.
Summary: This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of LNP.UCD.ABE in 5 pediatric subjects with severe infantile-onset UCDs. This is a master clinical protocol in which subjects with a variant in a urea cycle disorder (UCD) gene (CPS1, OTC, ASS1, ASL, ARG, NAGS, or SLC25A15) that is demonstrated...