Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)—10‐year follow‐up of patients who previously participated in an MPS VI survey study-Reference-Cited by-同舟云学术

Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)—10‐year follow‐up of patients who previously participated in an MPS VI survey study

Published:2014-04-24 Issue:8 Volume:164 Page:1953-1964
ISSN:1552-4825
Container-title:American Journal of Medical Genetics Part A
language:en
Short-container-title:Am. J. Med. Genet.

Author:

Giugliani Roberto¹,Lampe Christina²,Guffon Nathalie³,Ketteridge David⁴,Leão‐Teles Elisa⁵,Wraith James E.⁶,Jones Simon A.⁶,Piscia‐Nichols Cheri⁷,Lin Ping⁷,Quartel Adrian⁷,Harmatz Paul⁸

Affiliation:

1. Medical Genetics ServiceHCPADepartment of GeneticsUFRGSand INAGEMPPorto AlegreBrazil

2. Department of Pediatric and Adolescent MedicineVilla MetabolicaUniversity Medical Center of the University of MainzMainzGermany

3. Hôpital Femme Mère EnfantBronFrance

4. Women's and Children's HospitalNorth AdelaideAustralia

5. Hospital Pediátrico IntegradoCentro Hospitalar de S. JoãoPortoPortugal

6. Manchester Children's HospitalCentre for Genomic MedicineSt Mary's HospitalCMFTUniversity of ManchesterManchesterUnited Kingdom

7. BioMarin Pharmaceutical Inc.NovatoCalifornia

8. Children's Hospital & Research Center OaklandOaklandCalifornia

Funder

BioMarin Pharmaceutical Inc.

Publisher