(Q34281049)

English

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

scientific article

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

1 reference

Europe PubMed Central

Rare diseases and the assessment of intervention: what sorts of clinical trials can we use? (English)

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

1 reference

based on heuristic

inferred from title

author name string

Wilcken B

1

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

publication date

1 April 2001

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Journal of Inherited Metabolic Disease

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Europe PubMed Central

PubMed publication ID

1 August 2017

24

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

2

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

291-298

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

The natural history of homocystinuria due to cystathionine beta-synthase deficiency.

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Clinical trials and rare diseases: a way out of a conundrum

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Intelligence and quality of dietary treatment in phenylketonuria

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Methylmalonic Aciduria—A Newly Discovered Inborn Error

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Treatment of late-onset nonketotic hyperglycinaemia: effectiveness of imipramine and benzoate.

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Influence of phenylalanine intake on phenylketonuria

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Randomized versus historical controls for clinical trials.

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Randomized trials or observational tribulations?

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The natural history of untreated phenylketonuria over 20 years

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Proceedings of the 4th International Workshop of the Adrenoleukodystrophy International Research Group (ALD-IRG), University of York, 3 September 1998.

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Blood-brain phenylalanine relationships in persons with phenylketonuria

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Salient features of histidinemia

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Why "underpowered" trials are not necessarily unethical

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Therapy of X-linked adrenoleukodystrophy: prognosis based upon age and MRI abnormality and plans for placebo-controlled trials

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Dietary erucic acid therapy for X-linked adrenoleukodystrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Chronic renal failure in methylmalonic acidaemia

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Histidinaemia. Part III: Impact; a prospective study

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

"Underpowered" trials.

1 reference

https://pubmed.ncbi.nlm.nih.gov/11405347

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1023/A:1010387522195

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Dimensions Publication ID

0 references

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

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