Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates.

Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates.

Item
http://www.wikidata.org/entity/Q37021406
Recommendations and evidence for reporting items in pediatric clinical trial protocols and reports: two systematic reviewsA systematic review of approaches for engaging patients for research on rare diseasesClassification and natural history of the neuronal ceroid lipofuscinosesStandardized assessment of seizures in patients with juvenile neuronal ceroid lipofuscinosisRemote Assessment of Cognitive Function in Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease): A Pilot Study of Feasibility and Reliability.Involving patients in reducing decision uncertainties around orphan and ultra-orphan drugs: a rare opportunity?Future perspectives: Moving towards NCL treatments.Consensus on the criteria needed for creating a rare-disease patient registry. A Delphi study.Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review.An overview of the impact of rare disease characteristics on research methodology.Mitochondrial disease patient motivations and barriers to participate in clinical trials.Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.
P2860

Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates.

Item
http://www.wikidata.org/entity/Q37021406
description
article científic
@ca
article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
@tr
scientific article published on 26 April 2013
@en
vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Methodology of clinical resear ...... ration with patient advocates.
@en
Methodology of clinical resear ...... neuronal ceroid lipofuscinosis
@nl
type
label
Methodology of clinical resear ...... ration with patient advocates.
@en
Methodology of clinical resear ...... neuronal ceroid lipofuscinosis
@nl
prefLabel
Methodology of clinical resear ...... ration with patient advocates.
@en
Methodology of clinical resear ...... neuronal ceroid lipofuscinosis
@nl
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Methodology of clinical resear ...... ration with patient advocates.
@en
P2093
P2860
P304
P31
P356
P407
P433
P478
P577
P698
P921
P932