| Authors: |
Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Botha J; Takeda Pharmaceuticals International AG, Zurich, Switzerland. Electronic address: jaco.botha@takeda.com., Amartino H; Hospital Universitario Austral, Buenos Aires, Argentina., Giugliani R; Department of Genetics/UFRGS, Medical Genetics Service/HCPA, IMASP, DASA Genomica and Casa dos Raros, Porto Alegre, Brazil., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA., Kampmann C; Johannes Gutenberg University, Mainz, Germany., Link B; University Children's Hospital, Zurich, Switzerland., Lin SP; Mackay Memorial Hospital, Taipei, Taiwan., Molter D; Washington University School of Medicine, St. Louis, MO, USA., Raiman J; Birmingham Children's Hospital, NHS Foundation Trust, UK., Scarpa M; Udine University Hospital, Udine, Italy., Tylki-Szymańska A; Children's Memorial Health Institute, Warsaw, Poland., Jain S; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Whiteman DAH; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Burton BK; Ann & Robert H Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA. |
