(Q45324849)

13 December 2017

title

Clinical trials of orphan medicines (English)

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13 December 2017

Brendan M Buckley

1

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13 December 2017

language of work or name

English

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publication date

1 June 2008

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13 December 2017

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13 December 2017

volume

371

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13 December 2017

issue

9629

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13 December 2017

page(s)

2051-2055

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

13 December 2017

cites work

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Stiripentol in severe myoclonic epilepsy in infancy: a randomised placebo-controlled syndrome-dedicated trial. STICLO study group

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Bayesian clinical trials

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

The what, why and how of Bayesian clinical trials monitoring.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

N-of-1 trials: innovative methods to evaluate complementary and alternative medicines in pediatric cancer

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https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Predicting response to rhDNase and hypertonic saline in children with cystic fibrosis.

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https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Case studies, single-subject research, and N of 1 randomized trials: comparisons and contrasts

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https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Recombinant DNase in cystic fibrosis: a protocol for targeted introduction through n-of-1 trials. Scottish Cystic Fibrosis Group

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Clinical trials and rare diseases

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Sequential designs for phase III clinical trials incorporating treatment selection

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Sequelae of caffeine treatment in preterm infants with apnea

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Mitotane Use in Inoperable Adrenal Cortical Carcinoma

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

Chemotherapeutic management of the hormone-secreting endocrine malignancies

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2960876-4

7 January 2021

10.1016/S0140-6736(08)60876-4

Identifiers

DOI

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13 December 2017

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