Showing results (621-630 of 4,757) with videos related to

Sort By:
Pageof 476
Orphanet Journal of Rare Diseases|December 1, 2023
Self-reported functioning among patients with ultra-rare nemaline myopathy or a related disorder in Finland: a pilot studyVilma-Lotta Lehtokari, Minna Similä, Marianne Tammepuu, et al.
Orphanet Journal of Rare Diseases|December 1, 2023
Qualified placebo for trials of herbal medicine treatment in rare diseases? A cross-sectional analysisYixuan Li, Peipei Du, Xuebin Zhang, et al.
Orphanet Journal of Rare Diseases|November 29, 2023
Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022Jiayu Wu, Yang Yang, Jiaxin Yu, et al.
Orphanet Journal of Rare Diseases|January 14, 2024
Clinical, biochemical, and genetic spectrum of MADD in a South African cohort: an ICGNMD studyMichelle Bisschoff, Izelle Smuts, Marli Dercksen, et al.
Orphanet Journal of Rare Diseases|January 4, 2024
Hyperthermic intraperitoneal chemotherapy in patients with incomplete cytoreduction for appendiceal pseudomyxoma peritonei: a 10-year treatment experience in ChinaBing Wang, Ruiqing Ma, Guanjun Shi, et al.
Orphanet Journal of Rare Diseases|January 3, 2024
Implementation status of pharmacological studies in the development of orphan drugsSaki Yokoshiki, Teruyo Arato
Orphanet Journal of Rare Diseases|January 3, 2024
Dynamic monitoring of UBA1 somatic mutations in patients with relapsing polychondritisSuying Duan, Haiyang Luo, Yunchao Wang, et al.
Orphanet Journal of Rare Diseases|January 5, 2024
Design of mobile and website health application devices for drug tolerability in hereditary fructose intoleranceElsa Izquierdo-García, Andrea Lázaro-Cebas, Berta Montero Pastor, et al.
Orphanet Journal of Rare Diseases|January 3, 2024
Healthcare provider recognition of pregnancy related risks and management considerations in patients with tuberous sclerosis complexMeredith Rose, David Ritter, Nishant Gupta, et al.
Orphanet Journal of Rare Diseases|March 27, 2024
Moderate- to vigorous-intensity physical activities for hemophilia A patients during low-dose pharmacokinetic-guided extended half-life factor VIII prophylaxisChonlatis Srichumpuang, Arunothai Rakmanotham, Chatphatai Moonla, et al.
Pageof 476