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Expert Opinion on Orphan Drugs
Latest Publications
TOTAL DOCUMENTS
841
(FIVE YEARS 123)
H-INDEX
19
(FIVE YEARS 3)
Published By Informa Uk (Taylor & Francis)
2167-8707, 2167-8707
Latest Documents
Most Cited Documents
Contributed Authors
Related Sources
Related Keywords
Latest Documents
Most Cited Documents
Contributed Authors
Related Sources
Related Keywords
Lumasiran: expanding the treatment options for patients with primary hyperoxaluria type 1
Expert Opinion on Orphan Drugs
◽
10.1080/21678707.2021.2003779
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2021
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Author(s):
Sally-Anne Hulton
Keyword(s):
Treatment Options
◽
Primary Hyperoxaluria
◽
Primary Hyperoxaluria Type 1
◽
Primary Hyperoxaluria Type
◽
Hyperoxaluria Type
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Steroid alternatives for managing eosinophilic lung diseases
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.2003777
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2021
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Author(s):
Quentin Delcros
◽
Matthieu Groh
◽
Mouhamad Nasser
◽
Jean-Emmanuel Kahn
◽
Vincent Cottin
Keyword(s):
Lung Diseases
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An evaluation of onasemnogene abeparvovec for Spinal Muscular Atrophy (SMN1)
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.2003778
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2021
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Author(s):
Megan A. Waldrop
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Anne M. Connolly
◽
Jerry R. Mendell
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
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Efficacy of sirolimus for treatment of autoimmune lymphoproliferative syndrome: a systematic review of open label clinical studies
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1970523
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2021
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Author(s):
Shweta Sharma
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Md Sarfaraj Hussain
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Nidhi.B. Agarwal
◽
Dinesh Bhurani
◽
Mohd Ashif Khan
◽
...
Keyword(s):
Systematic Review
◽
Clinical Studies
◽
Autoimmune Lymphoproliferative Syndrome
◽
Open Label
◽
Lymphoproliferative Syndrome
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Patient experience in a rare disease: application of the IEXPAC questionnaire in hereditary transthyretin-mediated amyloidosis
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1953468
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2021
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Author(s):
Ana Peláez Bejarano
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Maria de las Aguas Robustillo Cortés
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Maria Isabel Guzman Ramos
Keyword(s):
Rare Disease
◽
Patient Experience
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European stakeholder perspectives on challenges to rare disease drug development – a qualitative study
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1953469
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2021
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Author(s):
Sarala Joshi
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Jessica Chen
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Mustafa Sultan
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Shefali Singh
◽
Syed Abedi
◽
...
Keyword(s):
Qualitative Study
◽
Drug Development
◽
Rare Disease
◽
Stakeholder Perspectives
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An evaluation of nifurtimox for Chagas disease in children
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1933431
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2021
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Author(s):
Fernanda Lascano
◽
J Altcheh
Keyword(s):
Chagas Disease
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Coenzyme Q10 and the exclusive club of diseases that show a limited response to treatment
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1932459
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2021
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Author(s):
Nadia Turton
◽
Nathan Bowers
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Sam Khajeh
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Iain P Hargreaves
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Robert A Heaton
Keyword(s):
Coenzyme Q10
◽
Response To Treatment
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Current and innovative therapeutic strategies for the treatment of giant cell arteritis
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1932458
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2021
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Author(s):
Alwin Sebastian
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Alessandro Tomelleri
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Bhaskar Dasgupta
Keyword(s):
Giant Cell Arteritis
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Giant Cell
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Therapeutic Strategies
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Old and novel prognostic biomarkers in primary biliary cholangitis
Expert Opinion on Orphan Drugs
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10.1080/21678707.2021.1927700
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2021
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Author(s):
G Mulinacci
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A Palermo
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Pietro Invernizzi
◽
Marco Carbone
Keyword(s):
Prognostic Biomarkers
◽
Primary Biliary Cholangitis
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