Pediatric Medical Geneticist, Pediatrician (Kids / Children Specialist)
Dr. Roberto Zori - Chief of Clinical Genetics and Metabolism/ Department of Pediatrics, Director of Cytogenetics; Interim Medical Director, Genetics/Molecular Pathology Laboratory Section; , Department of Pathology, Immunology and Laboratory, University of Forida;
Dr. Zori is a clinical geneticist and cytogeneticist. He is also a consultant to the craniofacial clinic for clinical genetics diagnostic and counseling issues
This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria鈥�
The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the鈥�
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