Results 181 to 190 of about 82,032 (316)

Real‐World Effectiveness and Safety of Pegzilarginase Treatment in Arginase 1 Deficiency

JIMD Reports, Volume 67, Issue 3, May 2026.
ABSTRACT Pegzilarginase is the first disease‐modifying drug for arginase 1 deficiency. In clinical trials, pegzilarginase effectively normalised plasma arginine (pArg), which was associated with clinically relevant improvements in neuromotor outcomes. We report from a French early access scheme the first report on experience from pegzilarginase therapy
Anaïs Brassier, Alina Arion, Jean‐Baptiste Arnoux, Magalie Barth, Dries Dobbelaere, Magali Gorce, Michael Grövdal, Cécile Laroche, Olivia Rime, Manuel Schiff, Mattias Rudebeck   +10 more
wiley   +1 more source

Detection of hemophilia carriers during pregnancy [PDF]

, 1982
LW Hoyer, CA Carta, MJ Mahoney
openalex   +1 more source

Radionuclides in the postoperative management of orthotopic human organ transplantation. [PDF]

, 1969
Brown, DW, Starzl, TE
core   +1 more source

Carrier screening in the reproductive setting—Are there medical implications for the heterozygote?—A guide for clinicians

Pregnancy, Volume 2, Issue 3, May 2026.
Abstract Carrier screening for genetic conditions performed preconception or during pregnancy allows identification of fetal risk for inherited autosomal recessive and X‐linked conditions. The goal is to identify at‐risk patients/couples and offer them reproductive options such as preimplantation genetic diagnosis, prenatal testing, or targeted newborn
Emily B. Rosenfeld, Nicole Kasatkin, Bi Liu Yu, Milen Velinov, Justin S. Brandt, Elena Ashkinadze   +5 more
wiley   +1 more source

An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study. [PDF]

Ther Adv Hematol
Babayeva S, Peyvandi A, Schepperle C, Auerswald G, Rauchfuss S, Peyvandi F, Cutica I, Miesbach W.   +7 more
europepmc   +1 more source

Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A [PDF]

, 2016
Johnny Mahlangu, Kazimierz Kuliczkowski, Faraizah Abdul Karim, Oleksandra Stasyshyn, Marina Kosinova, Lynda Mae Lepatan, Aleksander B. Skotnicki, Lisa Boggio, Robert Klamroth, Johannes Oldenburg, Andrzej Hellmann, Elena Santagostino, Ross Baker, Kathelijn Fischer, Joan Cox Gill, Stephanie P’ng, Pratima Chowdary, Miguel A. Escobar, Claudia Djambas Khayat, L. Rusen, Debra M. Bensen-Kennedy, Nicole Blackman, Tharin Limsakun, Alex Veldman, Katie St. Ledger, Ingrid Pabinger, for the AFFINITY Investigators   +26 more
openalex   +1 more source

Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review

British Journal of Pharmacology, Volume 183, Issue 9, Page 1779-1813, May 2026.
Abstract In the 2025 novel drug mini‐review, one can take a full measure of the ingenuity that underlies current drug design and development, despite the year's smaller harvest (46 novel drugs) compared to 2024 (53) and 2023 (70). 54% of the novel drugs are first‐in‐class (FIC).
Andreas Papapetropoulos, Stavros Topouzis, Steve P. H. Alexander, Miriam M. Cortese‐Krott, Zsuzsanna Helyes, Kirill Martemyanov, Claudio Mauro, Nithyanandan Nagercoil, Reynold A. Panettieri Jr, Hemal H. Patel, Rainer Schulz, Barbara Stefanska, Gary J. Stephens, Nathalie Vergnolle, Xin Wang, Stephen Ward, Péter Ferdinandy   +16 more
wiley   +1 more source

Emicizumab prophylaxis beyond clinical trials: a multicenter, prospective real-world study of pediatric hemophilia patients with and without inhibitors. [PDF]

Eur J Pediatr
Sherief LM, Elagamy O, Darwish A, Soliman NK, Elgendy MR, Nazim A, Sobhi A.   +6 more
europepmc   +1 more source

Strategies for Individualized Dosing of Clotting Factor Concentrates and Desmopressin in Hemophilia A and B [PDF]

, 2019
Tim Preijers, Lisette M. Schütte, Marieke J.H.A. Kruip, Marjon H. Cnossen, Frank W.G. Leebeek, Reinier M. van Hest, Ron A. A. Mathôt   +6 more
openalex   +1 more source

Redefining the role of the transfusion medicine physician in the era of advanced cellular therapies

Transfusion, EarlyView.
Eric A. Gehrie, Kevin J. Land
wiley   +1 more source