Expert Opinion on Orphan Drugs
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Steroid alternatives for managing eosinophilic lung diseases

2021 ◽  
Author(s):  
Quentin Delcros ◽  
Matthieu Groh ◽  
Mouhamad Nasser ◽  
Jean-Emmanuel Kahn ◽  
Vincent Cottin
Keyword(s):  
Lung Diseases

An evaluation of onasemnogene abeparvovec for Spinal Muscular Atrophy (SMN1)

2021 ◽  
Author(s):  
Megan A. Waldrop ◽  
Anne M. Connolly ◽  
Jerry R. Mendell
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy

Patient experience in a rare disease: application of the IEXPAC questionnaire in hereditary transthyretin-mediated amyloidosis

2021 ◽  
Author(s):  
Ana Peláez Bejarano ◽  
Maria de las Aguas Robustillo Cortés ◽  
Maria Isabel Guzman Ramos
Keyword(s):  
Rare Disease ◽  
Patient Experience

scholarly journals European stakeholder perspectives on challenges to rare disease drug development – a qualitative study

2021 ◽  
Author(s):  
Sarala Joshi ◽  
Jessica Chen ◽  
Mustafa Sultan ◽  
Shefali Singh ◽  
Syed Abedi ◽  
...  
Keyword(s):  
Qualitative Study ◽  
Drug Development ◽  
Rare Disease ◽  
Stakeholder Perspectives

scholarly journals Coenzyme Q10 and the exclusive club of diseases that show a limited response to treatment

2021 ◽  
Author(s):  
Nadia Turton ◽  
Nathan Bowers ◽  
Sam Khajeh ◽  
Iain P Hargreaves ◽  
Robert A Heaton
Keyword(s):  
Coenzyme Q10 ◽  
Response To Treatment

Old and novel prognostic biomarkers in primary biliary cholangitis

2021 ◽  
Author(s):  
G Mulinacci ◽  
A Palermo ◽  
Pietro Invernizzi ◽  
Marco Carbone
Keyword(s):  
Prognostic Biomarkers ◽  
Primary Biliary Cholangitis
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