Therapeutic advances in rare disease

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CiteScore	SJR	SNIP	CiteScore排名
2.0	0.447	0.662	学科排名百分位大类：Medicine 小类：Internal Medicine 102 / 167 39%

Therapeutic advances in rare disease - 最新文献

From roadmap to a sustainable end-to-end individualized therapy pathway.

Pub Date : 2025-05-27 DOI: 10.1177/26330040251339204 Anneliene H Jonker, Elena-Alexandra Tataru, David P Dimmock, Alison Bateman-House, Holm Graessner, Gareth Baynam, Erika F Augustine, Adam Jaffe, Anna M G Pasmooij, Oxana Iliach, Richard Horgan, James Davies, Shruti Mitkus, Larissa Lapteva, Matthis Synofzik, Timothy W Yu, Daniel O'Connor, Annemieke Aartsma-Rus

Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs.

Pub Date : 2025-04-28 DOI: 10.1177/26330040251335660 Nahya Awada

Navigating gynaecological challenges in Gaucher's disease: insights from five European countries.

Pub Date : 2025-04-28 DOI: 10.1177/26330040251335665 Ljubas Dominik, Krstulović Opara Anđela, Wagner Jasenka, Duraković Nadira

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