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Evidence evaluation in rare disease guidelines: a methodological perspective

Matt Bolz-Johnson, Thomas Kenny, Charlotte Gaasterland, Muhammad Imran Omar, Manon Engels, Agnies van Eeghen, Iris den Uijl, Willemijn Irvine
DOI: 10.20517/rdodj.2025.29 28 Oct 2025

Neurofibromatosis type 2-related schwannomatosis: an update

Donita Lightner
DOI: 10.20517/rdodj.2025.11 29 Oct 2025

A communication toolkit for the most impactful symptoms of Fabry disease: co-creation with Fabry disease patients and their treating clinicians in the UK

Derralynn Hughes, Sophie Thomas, Natalie Joseph-Williams, Katy Bunn, Stuart Gaffney, Kamran Iqbal, Tom Kenny
DOI: 10.20517/rdodj.2025.27 29 Oct 2025

Monoclonal gammopathies and their management in Gaucher disease type 1

 

Jeremy Lorber, Barry E Rosenbloom
DOI: 10.20517/rdodj.2025.30 28 Oct 2025

Management strategies for systemic lupus erythematosus using kinase inhibitors

Katie Liao, Hwai Jien Lee, Eric F Morand
DOI: 10.20517/rdodj.2025.08 3 Nov 2025

Emerging treatments for systemic lupus erythematosus

Yurie Satoh-Kanda, Shingo Nakayamada, Yoshiya Tanaka
DOI: 10.20517/rdodj.2025.45 3 Nov 2025

Evidence evaluation in rare disease guidelines: a methodological perspective

DOI: 10.20517/rdodj.2025.29
Matt Bolz-Johnson, Thomas Kenny, Charlotte Gaasterland, Muhammad Imran Omar, Manon Engels, Agnies van Eeghen, Iris den Uijl, Willemijn Irvine

Neurofibromatosis type 2-related schwannomatosis: an update

DOI: 10.20517/rdodj.2025.11
Donita Lightner
A communication toolkit for the most impactful symptoms of Fabry disease: co-creation with Fabry disease patients and their treating clinicians in the UK
DOI: 10.20517/rdodj.2025.27
Derralynn Hughes, Sophie Thomas, Natalie Joseph-Williams, Katy Bunn, Stuart Gaffney, Kamran Iqbal, Tom Kenny

Monoclonal gammopathies and their management in Gaucher disease type 1

 

DOI: 10.20517/rdodj.2025.30
Jeremy Lorber, Barry E Rosenbloom

Management strategies for systemic lupus erythematosus using kinase inhibitors

DOI: 10.20517/rdodj.2025.08
Katie Liao, Hwai Jien Lee, Eric F Morand

Emerging treatments for systemic lupus erythematosus

DOI: 10.20517/rdodj.2025.45
Yurie Satoh-Kanda, Shingo Nakayamada, Yoshiya Tanaka
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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