Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement.
Howie AH, Tingley K, Inbar-Feigenberg M, Mitchell JJ, Angel K, Gentle J, Smith M, Offringa M, Butcher NJ, Campeau PM, Chakraborty P, Chan A, Fergusson D, Mamak E, McClelland P, Mercimek-Andrews S, Mhanni A, Moazin Z, Rockman-Greenberg C, Rupar CA, Skidmore B, Stockler S, Thavorn K, Wyatt A, Potter BK; INFORM RARE Network.
Howie AH, et al. Among authors: thavorn k.
Orphanet J Rare Dis. 2024 Oct 23;19(1):393. doi: 10.1186/s13023-024-03364-x.
Orphanet J Rare Dis. 2024.
PMID: 39443985
Free PMC article.
Review.