Showing results (31-40 of 4,707) with videos related to

Sort By:
Pageof 471
Orphanet Journal of Rare Diseases|January 5, 2022
Impact of achondroplasia on Latin American patients: a systematic review and meta-analysis of observational studiesVirginia Fano, Chong A Kim, Pablo Rosselli, et al.
Orphanet Journal of Rare Diseases|January 5, 2022
Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018Min Fan, Adrienne Y L Chan, Vincent K C Yan, et al.
Orphanet Journal of Rare Diseases|January 20, 2022
Impact of the COVID-19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatmentAmanda Mortensen, Eva M Raebel, Samantha Wiseman
Orphanet Journal of Rare Diseases|January 7, 2022
Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trialsDeborah Elstein, Nadia Belmatoug, Patrick Deegan, et al.
Orphanet Journal of Rare Diseases|January 7, 2022
Cardiac device implantation and device usage in Fabry and hypertrophic cardiomyopathyRavi Vijapurapu, William Bradlow, Francisco Leyva, et al.
Orphanet Journal of Rare Diseases|January 9, 2022
The PID Life Index: an interactive tool to measure the status of the PID healthcare environment in any given countryLeire Solís, Julia Nordin, Johan Prevot, et al.
Orphanet Journal of Rare Diseases|March 1, 2019
Repurposing a rare opportunity: a brief insight into how implicit bias towards biomedicine impacts the care received by patients with a rare illnessLogan Zane John Williams
Orphanet Journal of Rare Diseases|March 21, 2021
Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveysTom Burke, Sohaib Asghar, Jamie O'Hara, et al.
Orphanet Journal of Rare Diseases|March 22, 2021
Diagnostic precision and identification of rare diseases is dependent on distance of residence relative to tertiary medical facilitiesAnna-Lena Walter, Florent Baty, Frank Rassouli, et al.
Orphanet Journal of Rare Diseases|March 22, 2021
The interoperability between the Spanish version of the International Classification of Diseases and ORPHAcodes: towards better identification of rare diseasesJuan Rico, Luis Javier Echevarría-González de Garibay, María García-López, et al.
Pageof 471