(Q35100707)

6 August 2017

title

Clinical research for rare disease: opportunities, challenges, and solutions (English)

1 reference

6 August 2017

0 references

Robert C Griggs

1 reference

6 August 2017

Mark Batshaw

1 reference

6 August 2017

Mary Dunkle

1 reference

6 August 2017

Rashmi Gopal-Srivastava

1 reference

6 August 2017

Edward Kaye

1 reference

6 August 2017

Jeffrey Krischer

1 reference

6 August 2017

Tan Nguyen

1 reference

6 August 2017

Kathleen Paulus

1 reference

6 August 2017

Peter A Merkel

1 reference

6 August 2017

Rare Diseases Clinical Research Network

1 reference

6 August 2017

publication date

13 November 2008

1 reference

Molecular Genetics and Metabolism

6 August 2017

published in

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6 August 2017

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6 August 2017

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6 August 2017

page(s)

20-26

1 reference

Challenges in the design and conduct of therapeutic trials in channel disorders

6 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva

9 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

Clinical trials and rare diseases

9 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

The ethics of placebo-controlled trials--a middle ground

9 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

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

9 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

9 July 2018

“Underpowered” trials

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

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

9 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

Applications of the case-control method. Overview and historical perspective.

9 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

Randomized versus historical controls for clinical trials.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3134795

A primer on the design, conduct, and interpretation of clinical trials

27 September 2018

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.003

Why “underpowered” trials are not necessarily unethical

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.003

Randomized trials or observational tribulations?

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.003

Determining optimal therapy--randomized trials in individual patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2008.10.003

Randomized phase II clinical trials

7 January 2021

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/19013090

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1016/J.YMGME.2008.10.003

1 reference

6 August 2017

1 reference

6 August 2017

1 reference