| Authors: |
Burton BK; Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA., Guffon N; Reference Center for Inherited Metabolic Diseases, Hospices Civils de Lyon, Lyon, France., Roberts J; Saint Mary's Hospital, Manchester, UK., van der Ploeg AT; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands., Jones SA; Saint Mary's Hospital, Manchester, UK., Audi J; Takeda Pharmaceuticals International AG, Zürich, Switzerland., Botha J; Takeda Pharmaceuticals International AG, Zürich, Switzerland. Electronic address: jaco.botha@takeda.com., Whiteman DAH; Takeda Pharmaceutical Company Limited, Lexington, MA, USA., Giugliani R; Postgraduate Program in Genetics and Molecular Biology UFRGS, Medical Genetics Service HCPA, INAGEMP, DASA and Casa dos Raros, Porto Alegre, Brazil., Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. |
