BOB体育

Skip to main content
(352) 294-5050

Roberto T Zori, MD

Pediatric Medical Geneticist, Pediatrician (Kids / Children Specialist)

Photo of Roberto T Zori
(352) 294-5050 MyUFHealth

Research at a glance

Top areas of exploration

  • Chromosome Deletion , 14 publications
  • Intellectual Disability , 13 publications
  • Syndrome , 13 publications
  • Phenotype , 11 publications

Research activity

101 publications

3,486 citations

Why is this important?

Active clinical trials

DFI17545

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�

Investigator
Roberto T Zori
Status
Accepting Candidates
Ages
18 Years - 65 Years
Sexes
All
DTX301-CL301
Investigator
Roberto T Zori
Status
Accepting Candidates
JNT-517

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�

Investigator
Roberto T Zori
Status
Accepting Candidates
Ages
18 Years - 65 Years
Sexes
All

My publications

101 publications

2024

Effects of oral sepiapterin on blood Phe concentration in a broad range of patients with phenylketonuria (APHENITY): results of an international, phase 3, randomised, double-blind, placebo-controlled trial.

Lancet (London, England)

鈥�

2024

Long-term comparative effectiveness of pegvaliase versus medical nutrition therapy with and without sapropterin in adults with phenylketonuria.

Molecular genetics and metabolism

鈥�

2024

Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program.

Molecular genetics and metabolism reports

鈥�

2023

Challenges and strategies for clinical trials in propionic and methylmalonic acidemias.

Molecular genetics and metabolism

鈥�

2023

Efficacy and safety of a synthetic biotic for treatment of phenylketonuria: a phase 2 clinical trial.

Nature metabolism

鈥�