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Adeno-associated viral vector gene therapy for canine hemophilia

Plexiform neurofibromas in neurofibromatosis type 1: current and emergent therapeutic strategies

Emerging treatments for systemic lupus erythematosus

Meeting Abstracts from the XVI International Collaboration on Rare Diseases and Orphan Drugs (ICORD): Incentivizing Science and a Comprehensive Program for Rare Diseases

Management strategies for systemic lupus erythematosus using kinase inhibitors

Educating hematologists about Gaucher disease

Neurofibromatosis type 2-related schwannomatosis: an update

Evidence evaluation in rare disease guidelines: a methodological perspective

Recognizing Gaucher disease in the fifth decade and beyond: a retrospective case study in patients of Ashkenazi Jewish descent

Monoclonal gammopathies and their management in Gaucher disease type 1

Reframing cutaneous neurofibromas as a model for extracellular matrix-driven benign tumor biology by R Brown

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