Therapeutic Advances in Rare Disease
SAGE Publications Ltd · United Kingdom · Est. 2020
Aims & Scope✦ Inferred from recent articles
This journal focuses on research and clinical advancements in rare diseases, including diagnosis, treatment development, patient outcomes, and healthcare access. Articles cover a range of rare conditions such as Fabry disease, Pompe disease, β-thalassemia, and IgA nephropathy, exploring novel therapies, gene therapies, enzyme replacement therapies, and patient-reported outcomes. The scope also extends to the operational aspects of rare disease research, including patient engagement, data registries, and drug access policies.
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