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Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement.

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Bibliographic Details
Title: Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement.
Authors: Howie, Alison H.1 (AUTHOR), Tingley, Kylie1 (AUTHOR), Inbar-Feigenberg, Michal2 (AUTHOR), Mitchell, John J.3 (AUTHOR), Angel, Kim4 (AUTHOR), Gentle, Jenifer5 (AUTHOR), Smith, Maureen6 (AUTHOR), Offringa, Martin7 (AUTHOR), Butcher, Nancy J.8 (AUTHOR), Campeau, Philippe M.9 (AUTHOR), Chakraborty, Pranesh10 (AUTHOR), Chan, Alicia11 (AUTHOR), Fergusson, Dean12 (AUTHOR), Mamak, Eva13 (AUTHOR), McClelland, Peyton10 (AUTHOR), Mercimek-Andrews, Saadet11 (AUTHOR), Mhanni, Aizeddin14,15 (AUTHOR), Moazin, Zeinab10 (AUTHOR), Rockman-Greenberg, Cheryl15 (AUTHOR), Rupar, C. Anthony16 (AUTHOR)
Source: Orphanet Journal of Rare Diseases. 10/23/2024, Vol. 19 Issue 1, p1-15. 15p.
Database: Academic Search Ultimate
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Description
ISSN:17501172
DOI:10.1186/s13023-024-03364-x