(Q91974053)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

review article

1 reference

title

Therapies for rare diseases: therapeutic modalities, progress and challenges ahead (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

26 April 2020

corrigendum / erratum

0 references

author

Kay Davies

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

Tudor I Oprea

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

Marco Prunotto

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

9

Stephen C Groft

1 reference

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26 April 2020

Robert Preti

10

object named as

Robert Preti

1 reference

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26 April 2020

Katherine I Miller Needleman

6

object named as

Katherine I Miller Needleman

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

author name string

Erik Tambuyzer

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

Benjamin Vandendriessche

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

Christopher P Austin

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

Philip J Brooks

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

Kristina Larsson

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

James Valentine

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

publication date

13 December 2019

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

Nature Reviews Drug Discovery

26 April 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

volume

19

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

issue

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

page(s)

93-111

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

Rare diseases, orphan drugs and their regulation: questions and misconceptions

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Pharos: Collating protein information to shed light on the druggable genome

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eRAM: encyclopedia of rare disease annotations for precision medicine

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7 January 2021

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7 January 2021

[Strategies for digitalizing the hospital of the future]

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7 January 2021

Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data

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7 January 2021

Usefulness of an Implantable Loop Recorder to Detect Clinically Relevant Arrhythmias in Patients With Advanced Fabry Cardiomyopathy

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7 January 2021

Amount and intensity of daily living activities in Charcot-Marie-Tooth 1A patients

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7 January 2021

Machine Learning to Improve Energy Expenditure Estimation in Children With Disabilities: A Pilot Study in Duchenne Muscular Dystrophy

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7 January 2021

The haemtrack home therapy reporting system: Design, implementation, strengths and weaknesses: A report from UK Haemophilia Centre Doctors Organisation.

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7 January 2021

Innovative approach for self-management and social welfare of children with cystic fibrosis in Europe: development, validation and implementation of an mHealth tool (MyCyFAPP).

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7 January 2021

Patient reported outcome measures in rare diseases: a narrative review.

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7 January 2021

Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report

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7 January 2021

Home Monitoring of Patients with Cystic Fibrosis to Identify and Treat Acute Pulmonary Exacerbations. eICE Study Results

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7 January 2021

The art and science of choosing efficacy endpoints for rare disease clinical trials

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7 January 2021

Preparing for the Future of Rare Diseases

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7 January 2021

Impact of remote patient monitoring on clinical outcomes: an updated meta-analysis of randomized controlled trials

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7 January 2021

Non-invasive delivery strategies for biologics

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7 January 2021

10.1038/S41573-019-0049-9

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:31836861%20AND%20SRC:MED&resulttype=core&format=json

26 April 2020

1 reference