Results 241 to 250 of about 3,868,024 (353)

Orphan drugs policies: a suitable case for treatment [PDF]

, 2014
Michael Drummond, Adrian Towse
openalex   +1 more source

The photochemical inheritance of Eduardo Lissi and Juan Grotewold and the intersystem crossings with other inheritances

Photochemistry and Photobiology, EarlyView.
Eduardo Lissi, Dorita Adamo‐Lissi and Juan Grotewold in Aberystwyth, Wales, winter 1961. Upon their return to Buenos Aires in 1963, with their PhD degrees, Lissi and Grotewold started a Chemical Kinetics and Photochemistry group in the School of Sciences (University of Buenos Aires).
Silvia E. Braslavsky, Carlos M. Previtali   +1 more
wiley   +1 more source

Adverse Reactions to the Orphan Drug Cerliponase Alfa in the Treatment of Neurolipofuscinosis Type 2 (CLN2). [PDF]

Pharmaceuticals (Basel)
Ammendolia I, Sframeli M, Esposito E, Cardia L, Noto A, Currò M, Calapai G, De Pasquale M, Mannucci C, Calapai F.   +9 more
europepmc   +1 more source

Reimbursement of orphan drugs in Belgium: what (else) matters? [PDF]

, 2014
Eline Picavet, David Cassiman, Steven Simoens   +2 more
openalex   +1 more source

The effect of leukoreduction and prolonged storage on coagulation in cold‐stored whole blood: An in vitro study

Vox Sanguinis, EarlyView.
Abstract Background and Objectives Cold‐stored whole blood (CSWB) for haemodynamically unstable bleeding patients is especially convenient when platelets are otherwise unavailable and simple logistics are favoured, as in prehospital situations. Storage times exceeding the currently common 21 days would be beneficial in situations where normal blood ...
Sanna Susila, Teemu Silver, Tuukka Helin, Timo Jama, Jouni Lauronen, Lotta Joutsi‐Korhonen, Minna Ilmakunnas   +6 more
wiley   +1 more source

Successes and pitfalls in orphan drug development for sickle cell disease. [PDF]

Blood Adv
Costa E, Isgrò A, de Montalembert M, Leufkens HGM, Ware RE, De Franceschi L.   +5 more
europepmc   +1 more source

Effectiveness of Pyridoxal‐5′‐Phosphate in PNPO Deficiency: A Systematic Review

Journal of Inherited Metabolic Disease, Volume 48, Issue 5, September 2025.
ABSTRACT Pyridox(am)ine 5′‐phosphate oxidase (PNPO) deficiency is an ultrarare inherited neurometabolic disease, characterized by primarily neonatal‐onset B6‐responsive epileptic encephalopathies. Treatment often requires sustainable access to high‐quality pyridoxal‐5′‐phosphate (PLP, i.e., active vitamin B6), although some patients (also) respond to ...
Nina N. Stolwijk, Laura van Dussen, Niels D. Reijnhout, Marion M. M. G. Brands, Bregje Jaeger, Peter T. Clayton, Carla E. M. Hollak, Annet M. Bosch   +7 more
wiley   +1 more source

Navigating health policies and programs in India: exploring opportunities to improve rare disease management and orphan drug research. [PDF]

Orphanet J Rare Dis
Mishra S, Bhat D, Venkatesh MP.
europepmc   +1 more source

Repurposing the orphan drug nitisinone to control the transmission of African trypanosomiasis. [PDF]

PLoS Biol, 2021
Sterkel M, Haines LR, Casas-Sánchez A, Owino Adung'a V, Vionette-Amaral RJ, Quek S, Rose C, Silva Dos Santos M, García Escude N, Ismail HM, Paine MI, Barribeau SM, Wagstaff S, MacRae JI, Masiga D, Yakob L, Oliveira PL, Acosta-Serrano Á.   +17 more
europepmc   +1 more source

Reimbursement of orphan drugs: the Pompe and Fabry case in the Netherlands [PDF]

, 2014
Rinke van den Brink
openalex   +1 more source