Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)

Roberto Giugliani

Porto Alegre, RS, BR 
Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Porto Alegre, RS, BR 
OverviewLocationsClinical Research

Overview

Roberto Giugliani practices in Porto Alegre, Brazil. Mr. Giugliani and is rated as an Elite expert by MediFind in the treatment of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome). His top areas of expertise are Mucopolysaccharidoses (MPS), Mucopolysaccharidosis Type 2 (MPS II, Hunter Syndrome), Acid Sphingomyelinase Deficiency (ASMD), Mucopolysaccharidosis Type 7 (MPS VII, Sly Syndrome), and Bone Marrow Transplant.

His clinical research consists of co-authoring 378 peer reviewed articles and participating in 18 clinical trials. MediFind looks at clinical research from the past 15 years. In particular, he has co-authored 31 articles and participated in 1 clinical trial in the study of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome).

Gender
Male

Locations

Porto Alegre, RS, Brazil
Other Locations
Sao Paulo, SP, Brazil
Rio Grande, RS, Brazil
Brazil

Clinical Research

Clinical research consists of overseeing clinical studies of patients undergoing new treatments and therapies, and publishing articles in peer reviewed medical journals. Experts who actively participate in clinical research are generally at the forefront of the fields and aware of the most up-to-date advances in treatments for their patients.

18 Clinical Trials

A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
Enrollment Status: Active_not_recruiting
Publish Date: March 07, 2025
Intervention Type: Drug
Study Drug: SHP611
Study Phase: Phase 2
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Enrollment Status: Active_not_recruiting
Publish Date: January 28, 2025
Intervention Type: Genetic
Study Phase: Phase 2/Phase 3
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
Enrollment Status: Terminated
Publish Date: December 30, 2024
Intervention Type: Drug
Study Drug: Venglustat GZ402671
Study Phase: Phase 3
A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects With Fabry Disease
A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects With Fabry Disease
Enrollment Status: Terminated
Publish Date: June 17, 2024
Intervention Type: Drug
Study Drug: Replagal
Study Phase: Phase 3
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type I
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type I
Enrollment Status: Active_not_recruiting
Publish Date: December 06, 2023
Intervention Type: Genetic
Study Phase: Phase 1/Phase 2
Biomarker for Gilbert Disease - An International, Multi- Center, Epidemiological Protocol
Biomarker for Gilbert Disease - An International, Multi- Center, Epidemiological Protocol
Enrollment Status: Withdrawn
Publish Date: February 13, 2023
Biomarker for Niemann Pick Type C Disease (NPC1/NPC2) an International, Multicenter, Epidemiological Study
Biomarker for Niemann Pick Type C Disease (NPC1/NPC2) an International, Multicenter, Epidemiological Study
Enrollment Status: Withdrawn
Publish Date: February 13, 2023
Biomarkers for Hunter Syndrome: An International, Multicenter, Observational, Longitudinal Protocol
Biomarkers for Hunter Syndrome: An International, Multicenter, Observational, Longitudinal Protocol
Enrollment Status: Terminated
Publish Date: February 10, 2023
A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Enrollment Status: Unknown
Publish Date: December 19, 2022
Intervention Type: Drug
Study Drug: Losartan
Study Phase: Phase 2
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Enrollment Status: Active_not_recruiting
Publish Date: October 26, 2022
A Retrospective and Cross-sectional Study to Evaluate Neurodevelopmental Status in Pediatric Subjects With Severe Mucopolysaccharidosis Type II (Hunter Syndrome)
A Retrospective and Cross-sectional Study to Evaluate Neurodevelopmental Status in Pediatric Subjects With Severe Mucopolysaccharidosis Type II (Hunter Syndrome)
Enrollment Status: Completed
Publish Date: April 26, 2022
An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Enrollment Status: Completed
Publish Date: August 31, 2021
A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
Enrollment Status: Completed
Publish Date: June 10, 2021
Intervention Type: Biological
Study Phase: Phase 2/Phase 3
An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
Enrollment Status: Completed
Publish Date: June 10, 2021
Intervention Type: Biological
Study Phase: Phase 2/Phase 3
A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy
A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy
Enrollment Status: Completed
Publish Date: June 08, 2021
Intervention Type: Biological
Study Phase: Phase 4
A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
Enrollment Status: Completed
Publish Date: June 08, 2021
Intervention Type: Biological
A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease
A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease
Enrollment Status: Completed
Publish Date: October 31, 2018
Intervention Type: Drug
Study Phase: Phase 2
A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease
A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease
Enrollment Status: Completed
Publish Date: October 03, 2018
Intervention Type: Drug
Study Phase: Phase 2
View 17 Less Clinical Trials

378 Total Publications

Practical recommendations for diagnosis, management, and follow-up of Niemann-Pick type-C disease patients: a Brazilian perspective.
Practical recommendations for diagnosis, management, and follow-up of Niemann-Pick type-C disease patients: a Brazilian perspective.
Journal: Arquivos de neuro-psiquiatria
Published: May 09, 2025

Areas of Expertise

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