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

[1]  H. Levy,et al.  Histidinaemia. Part III: Impact; a prospective study , 1983, Journal of Inherited Metabolic Disease.

[2]  W. Köhler,et al.  Proceedings of the 4th International Workshop of the Adrenoleukodystrophy International Research Group (ALD-IRG), University of York, 3 September 1998 , 2000, Journal of Inherited Metabolic Disease.

[3]  H. Moser,et al.  Therapy of X-linked adrenoleukodystrophy: Prognosis based upon age and MRI abnormality and plans for placebo-controlled trials , 2000, Journal of Inherited Metabolic Disease.

[4]  E. Wiltshire,et al.  Treatment of late-onset nonketotic hyperglycinaemia: Effectiveness of imipramine and benzoate , 2000, Journal of Inherited Metabolic Disease.

[5]  B. Wilcken,et al.  Neonatal diagnosis of long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency and implications for newborn screening by tandem mass spectrometry , 1999, Journal of Inherited Metabolic Disease.

[6]  T. Barratt,et al.  Chronic renal failure in methylmalonic acidaemia , 2004, European Journal of Pediatrics.

[7]  R. Moats,et al.  Blood–Brain Phenylalanine Relationships in Persons With Phenylketonuria , 2000, Pediatrics.

[8]  S J Pocock,et al.  Randomized trials or observational tribulations? , 2000, The New England journal of medicine.

[9]  R. Hogg,et al.  Triple-combination antiretroviral therapy in sub-Saharan Africa , 1997, The Lancet.

[10]  M. Griffiths “Underpowered” trials , 1997, The Lancet.

[11]  David Braunholtz,et al.  Why “underpowered” trials are not necessarily unethical , 1997, The Lancet.

[12]  J G Thornton,et al.  Clinical trials and rare diseases: a way out of a conundrum , 1995, BMJ.

[13]  D. Danks,et al.  The natural history of untreated phenylketonuria over 20 years , 1991, Journal of paediatrics and child health.

[14]  A. Ades,et al.  Intelligence and quality of dietary treatment in phenylketonuria. , 1990, Archives of disease in childhood.

[15]  W. Rizzo,et al.  Dietary erucic acid therapy for X‐linked adrenoleukodystrophy , 1989, Neurology.

[16]  K. Pettigrew,et al.  The natural history of homocystinuria due to cystathionine beta-synthase deficiency. , 1985, American journal of human genetics.

[17]  T C Chalmers,et al.  Randomized versus historical controls for clinical trials. , 1982, The American journal of medicine.

[18]  L. Barness,et al.  Methylmalonic aciduria. A newly discovered inborn error. , 1968, Annals of internal medicine.

[19]  M. Partington,et al.  Salient features of histidinemia. , 1967, American journal of diseases of children.

[20]  H. Bickel,et al.  Influence of phenylalanine intake on phenylketonuria. , 1953, Lancet.