Rare diseases and the assessment of intervention: What sorts of clinical trials can we use?-Reference-Cited by-同舟云学术

Rare diseases and the assessment of intervention: What sorts of clinical trials can we use?

Published:2001-04 Issue:2 Volume:24 Page:291-298
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Wilcken B.¹

Affiliation:

1. ; The Children's Hospital at Westmead; Locked Bag 4001 Westmead, New South Wales 2145, (Sydney) Australia

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1023/A:1010387522195/fullpdf

Reference23 articles.

1. The mutational spectrum in the MCAD gene of newborns identi¢ed by prospective tandem MS screening for ‘diagnostic’ acyl-carnitines in blood spots differs from that observed in clinically a¡ected patients;Andresen;J Inherit Metab Dis,2000

2. Proceedings of the 4th Inter-nationalWorkshop of the Adrenolekodystrophy International Research Group, September 1998;Alger;J. Inherit Metab Dis,2000

3. In£uence of phenylalanine intake on phenylketonuria;Bickel;Lancet,1953

4. Newborn screening for MCAD de¢ciency: the New South Walse experience;Carpenter;J Inherit Metab Dis,2000

5. Histidinaemia part III: Impact: a prospective study;Coulombe;J Inher Metab Dis,1983

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