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

Paving the way toward treatment solutions for CTNNB1 syndrome: a patient organization perspective.

Pub Date : 2025-02-12 DOI: 10.1177/26330040251318355 Špela Miroševič, Shivang Khandelwal, Emily Amerson, Effie Parks, Mariana Parks, Lauren Cochran, Ana González Hernández, Mirela Ferraro, Leszek Lisowski, Andrea Perez-Iturralde, Wendy Chung, Michele H Jacob, Nina Žakelj, Duško Lainšček, Vida Forstnerič, Petra Sušjan, Matea Maruna, Roman Jerala, Damjan Osredkar

Nirogacestat-the pathway to approval of the first treatment for desmoid tumors, a rare disease.

Pub Date : 2025-02-11 DOI: 10.1177/26330040251317546 Shivaani Kummar, Nam Bui, Wells A Messersmith, Jeanne Whiting, Marlene Portnoy, Allison Lim, L Mary

Behcet's disease in a tertiary eye hospital in Pakistan.

Pub Date : 2025-01-18 DOI: 10.1177/26330040251314126 Tabish Ali Shalwani, Alishan Khowaja, Narmeen Punjwani

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