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The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases.
Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.
Orphanet J Rare Dis. 2021.
PMID: 33874971
Free PMC article.
Review.
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Sidman M, Kakkis ED, Cox GF.
Clarke LA, et al.
Pediatrics. 2009 Jan;123(1):229-40. doi: 10.1542/peds.2007-3847.
Pediatrics. 2009.
PMID: 19117887
Clinical Trial.
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