Results 171 to 180 of about 88,754 (289)

Incidence of systemic lupus erythematosus in patients with immune thrombocytopenia: A nationwide study

British Journal of Haematology, EarlyView.
Malin Rasmussen, Nikolaj Mannering, Henrik Frederiksen   +2 more
wiley   +1 more source

Efficacy and Safety of Prophylaxis With a Plasma‐Derived von Willebrand Factor/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease—A WIL‐31 Study Sub‐Analysis

European Journal of Haematology, EarlyView.
ABSTRACT Objectives The WIL‐31 study demonstrated efficacy and safety of prophylaxis with the plasma‐derived von Willebrand factor/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on‐demand run‐in study as an intra‐individual comparator.
Claudia Djambas Khayat, Leonid Dubey, Adlette Inati, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Robert F. Sidonio Jr, Ali T. Taher, Kateryna V. Vilchevska, Vladimir Vdovin, Ana Boban   +10 more
wiley   +1 more source

Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion. [PDF]

Health Expect
Frost J, Widening Engagement Patient Advisory Group, Mandizha J, van den Dungen C, Asare L, Pope C.   +5 more
europepmc   +1 more source

Current status and trend of clinical development of orphan drugs in China. [PDF]

Orphanet J Rare Dis, 2022
Xiang Z, Jiang W, Yan B, Jiang J, Zheng H.   +4 more
europepmc   +1 more source

Bexicaserin for the treatment of seizures in developmental and epileptic encephalopathies: A phase 1b/2a trial (PACIFIC)

Epilepsia, EarlyView.
Abstract Objective This randomized, double‐blind, phase 1b/2a clinical trial was designed to evaluate the safety, tolerability, and efficacy of oral bexicaserin versus placebo for the treatment of seizures in adolescents and adults with developmental and epileptic encephalopathies (DEEs).
Dennis J. Dlugos, Ingrid E. Scheffer, Jacqueline A. French, David G. Vossler, Chad Orevillo, Shikha Polega, Randall Kaye, and the LP352‐201 Study Investigators, Kore Liow, Ena Zhu, Jennifer Waldron, Hayley Groubert, Alexis Anderson, Pavel Klein, Salman Hashmi, Shicong Ye, Anita Farhi, David Vossler, Dione M. Froman, Shannon L. Hoffman‐Huffaker, Ki Hyeong Lee, AnnaLyn Dallas, Amir Arain, Laura Beeler, Michael Macken, Irena Bellinski, Elizabeth Cunningham, James Renfroe, Shezsay Colbert, Robert Hogan, Patty Schaefer, Susan Shaw, Sheetal Desai, Ahmed Sadek, Haile Robertson, Kamil Detyniecki, Anabela Cieslicki, Ruben Kuzniecky, Sarah Goodman, Selim Benbadis, Marina Azevedo, Sami Aboumatar, Julie Biel, Nilika Singhal, Emerald Wan, Evan Fertig, Hannah Hart, Debopam Samanta, Sarah Hankins, Steven Wolf, Patricia Pena, Robert Nahouraii, M. J. Mari, David Burdette, Stephanie Mueller, Karen Keough, Victoria Henderson, Talitha Durgin, Terence O’Brien, Rafael Smith, Jack Germaine, Kate Riney, Lori Anthony, David Reutens, Kimberley Irwin, Michael Fahey, Melissa Batt, Stephanie Kaladis, Kasthoori Jeyachanthiran, Ingrid Scheffer, Rebecca Wharrie, Greesha Zacharia, Brittany Walsh, Heather Robinson, Sharon Ganter   +74 more
wiley   +1 more source

Methodological challenges in Dutch HTA of non-oncological orphan drugs: a retrospective analysis and price comparison using different pricing models. [PDF]

Orphanet J Rare Dis
Walraven J, Kaveh M, Uyl-de Groot C.
europepmc   +1 more source

Societal preferences for funding orphan drugs in China: An application of the discrete choice experiment method. [PDF]

Front Public Health, 2022
Tan S, Wang Y, Tang Y, Jiang R, Chen M, Chen H, Yang F.   +6 more
europepmc   +1 more source

Lennox–Gastaut syndrome unveiled: Advancing diagnosis, therapies, and advocacy‐insights from the Genoa International Workshop

Epilepsia, EarlyView.
Abstract Lennox–Gastaut syndrome (LGS) is one of the most severe, yet one of the most discussed, childhood‐onset developmental and epileptic encephalopathies (DEEs). Dissent among epileptologists on the definition and minimum set of electroclinical features derives from the high etiological heterogeneity within the syndrome, which could make its ...
Antonella Riva, Gianluca D'Onofrio, Elisabetta Amadori, Alexis Arzimanoglou, Stéphane Auvin, Irene Bagnasco, Paola Barabino, Valentina Biagioli, Isabella Brambilla, Giuliana Cangemi, Antonietta Coppola, Antonella De Lillo, Carlo Di Bonaventura, Giancarlo Di Gennaro, Edoardo Ferlazzo, Antonio Gil‐Nagel, Giuseppe Gobbi, Simona Lattanzi, Gerhard Kluger, Günter Krämer, Maria Margherita Mancardi, Carlo Minetti, Lino Nobili, Elisa Paravati, Milka Pringsheim, Erika Rebessi, Antonino Romeo, Angelo Russo, Emilio Russo, Katia Santoro, Susanne Schubert‐Bast, Laura Siri, Jo Sourbron, Maria Stella Vari, Alberto Verrotti, Flavio Villani, Maurizio Viri, Celina von Stülpnagel, Nelia Zamponi, Federico Zara, Pasquale Striano   +40 more
wiley   +1 more source

Establishment of a value assessment framework for orphan drugs in China: an application of the discrete choice experiment in multicriteria decision analysis. [PDF]

Front Pharmacol
Zhao Z, Tang X, Hu M.
europepmc   +1 more source

Criteria to define rare diseases and orphan drugs: a systematic review protocol. [PDF]

BMJ Open, 2022
Abozaid GM, Kerr K, McKnight A, Al-Omar HA.   +3 more
europepmc   +1 more source