Strategic Drug Development for Rare and Underexplored Diseases

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REVIEW

Published on 22 Aug 2025

Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches

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Sofea ‘Aisyah Aminondin
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Noor Afiqah Jasmadi
Nur Irdeena Nabella Nizam
Ilhan Syahmi Shahrul
Deny Susanti
Junaidi Khotib
Md Faiyazuddin
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Muhammad Salahuddin Haris

doi 10.3389/fphar.2025.1630994

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ORIGINAL RESEARCH

Published on 20 Aug 2025

Safety assessment of laronidase: real-world adverse event analysis based on the FDA adverse event reporting system (FAERS)

Zhuomiao Lin
Junling Xue
Meiqing Yang
Xihui Yu
Jiahong Zhong

doi 10.3389/fphar.2025.1623921

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Frontiers in Pharmacology

Frontiers in Pharmacology

Experimental Pharmacology and Drug Discovery

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