DrRoberto Zori

A Phase 3, Double-Blind, Randomized, Two-Period, Multicenter, Placebo-Controlled, Efficacy and Safety Study of JNT-517 for the Treatment of Participants with Phenylketonuria

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants with Phenylketonuria

A PHASE 2 Study of JNT-517 in Adolescent Participants with Phenylketonuria

“A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of mRNA 3210 in Participants with Phenylketonuria” (“Study”) in accordance with Sponsor’s protocol no. mRNA-3210-P101 (“Protocol”); - Start Up Fees

A Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria (OPAL)

A Phase 1/Phase 2, open-label, dose-escalation, and dose expansion study to evaluate the safety, tolerability, and efficacy of SAR444836, an adeno-associated viral vector-mediated gene transfer of human phenylalanine hydroxylase, in adult participants with phenylketonuria

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8 (AAV8)-mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late- onset OTC Deficiency

A Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of SYNB1934 in Patients with PKU (SYNPHENY-3)

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

A PHASE 3 OPEN-LABEL STUDY OF SAFETY OF WEEKLY SUBCUTANEOUS PEGZILARGINASE IN SUBJECTS WITH ARGINASE I DEFICIENCY

A Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria

A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design

A PHASE 3 STUDY OF PTC923 IN SUBJECTS WITH PHENYLKETONURIA

A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients with Ornithine Transcarbamylase Deficiency

An Open-label Study of the Efficacy and Safety of SYNB1618 and SYNB1619 in Patients with Phenylketonuria (SynPheny-1)

A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults with Arginase 1 Deficiency

A Prospective Clinical Study of Phenylketonuria (PKU)

A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care

HPN-100-021: A Randomised, Controlled, Open-Label Parallel Arm Study of the Safety, Pharmacokinetics and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate [GPB]) Oral Liquid and Sodium Phenylbutyrate (NaPBA) in Phenylbutyrate Treatment Naïve Patients with Urea Cycle Disorders

An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/day Dose) in Adults with Phenylketonuria

A Non-Interventional Observational Study of Pegvaliase-Naïve Adults with Phenylketonuria (PKU): Concept Elicitation and Cognitive Interviews

An Open-label, Multicenter Extension Study To Evaluate The Long-Term Safety, Tolerability And Effects Of Intravenous AEB1102 In Patients with Arginase I Deficiency Who Previously Received Treatment In Study CAEB1102-101A

A phase 2 Adult Angelman Syndrome Clinical Trial: A Randomized, Double-blind, Safety and Efficacy of Gaboxadol

CMS Newborn Screening - COQWX