rare diseases

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1
Estimating the fiscal impact of rare diseases using a public economic framework: a case study applied to hereditary transthyretin-mediated (hATTR) amyloidosis.
Connolly, Mark P;Panda, Saswat;Patris, Julien;Hazenberg, Bouke P C;
2019 orphanet journal of rare diseases DOI: 10.1186/s13023-019-1... 308 views
2
European lipodystrophy registry: background and structure.
von Schnurbein, Julia;Adams, Claire;Akinci, Baris;Ceccarini, Giovanni;D'Apice, Maria Rosaria;Gambine...
2020 orphanet journal of rare diseases DOI: 10.1186/s13023-020-1... 194 views
3
pemphigus herpetiformis - a case report of a rare form of pemphigus and review of the literature
;Stojković-Filipović Jelena;Lekić Branislav;Milčić Danijela;Milinković Mirjana V.
2016 applied nursing research : anr DOI: 10.2298/VSP150617044... 139 views
4
computer-assisted initial diagnosis of rare diseases
;Rui Alves;Marc Piñol;Jordi Vilaplana;Ivan Teixidó;Joaquim Cruz;Jorge Comas;Ester Vilaprinyo;Albert...
2016 pediatrics DOI: 10.7717/peerj.2211 197 views
5
incidence and prevalence of systemic sclerosis (ssc) in poland – differences between rural and urban regions
;Krzysztof Kanecki;Paweł Goryński;Patryk Tarka;Waldemar Wierzba;Piotr Tyszko
2017 biomedicine and pharmacotherapy DOI: 10.5604/12321966.123... 159 views
6
the german government's global health strategy – a strategy also to support research and development for neglected diseases?
;Angela Fehr;Oliver Razum
2014 sustainability (switzerland) DOI: 10.3402/gha.v7.24565 179 views

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fiscal analysis generational accounting hereditary transthyretin-derived (hattr) amyloidosis public economics Registry adipose tissue lipodystrophy diagnosis drug therapy treatment outcomemedicine (general) eHealth Epidemiology global health neglected diseases