Condition

Mucopolysaccharidosis Type 7

Find a Doctor

Roberto Giugliani

Hospital De Clínicas De Porto Alegre
Male

Bio


Roberto Giugliani is in Porto Alegre, Brazil. Dr. Giugliani is highly experienced in treating patients with Mucopolysaccharidosis Type 7. They have been an author on 361 peer reviewed articles and participated in 5 clinical trials in the past 15 years. In particular, they have been an author on 4 peer reviewed articles regarding Mucopolysaccharidosis Type 7.

Contact

Porto Alegre, RS 90035, BR

Latest Advances


Latest Advance
Study
  • Condition: Severe Mucopolysaccharidosis Type I
  • Journal: Orphanet journal of rare diseases
  • Treatment Used: Intravenous Brain-penetrating Insulin Receptor Antibody-iduronidase Fusion Protein
  • Number of Patients: 11
  • Published —
In this study, researchers evaluated the outcomes of having 1 year of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein in children with severe Mucopolysaccharidosis Type I.
Latest Advance
Study
  • Condition: Fabry disease
  • Journal: Orphanet journal of rare diseases
  • Treatment Used: Migalastat
  • Number of Patients: 50
  • Published —
This study evaluated the use of migalastat, an orally-administered small molecules for patients with Fabry disease who experience gastrointestinal symptoms, including diarrhea.

Clinical Trials


Clinical Trial
  • Status: Recruiting
  • Participants: 40
  • Start Date: June 12, 2020
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 42
  • Start Date: April 30, 2019
A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 30
  • Start Date: November 7, 2018
A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Genetic
  • Participants: 12
  • Start Date: September 27, 2018
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Clinical Trial
  • Status: Recruiting
  • Participants: 60
  • Start Date: November 16, 2017
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

All Publications
View All


Publication
Study
  • Journal: International journal of neonatal screening
  • Published —
Neonatal Screening for MPS Disorders in Latin America: A Survey of Pilot Initiatives.
Publication
Study
  • Journal: Orphanet journal of rare diseases
  • Published —
Estimated prevalence of mucopolysaccharidoses from population-based exomes and genomes.

Contact

Porto Alegre, RS 90035, BR

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