RARE DISEASE AND ORPHAN DRUGS JOURNAL
OAE PUBLISHING INC · US · Est. 2021
Aims & Scope✦ Inferred from recent articles
The journal focuses on rare diseases and orphan drugs, covering genetic disorders such as Gaucher disease, muscular dystrophies (Duchenne), neurofibromatosis type 1, and hemophilia. It also addresses systemic autoimmune diseases like lupus erythematosus and rare neurological conditions. Research includes disease mechanisms, diagnostic tools, therapeutic strategies like gene therapy and stem cell therapy, and clinical trial advancements.
AI-summarised from recent articles · verify on the publisher page
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