Results 141 to 150 of about 68,267 (269)

PB2645: HEMATURIA IN ADULT HAEMOPHILIC INDIVIDUALS: IS THE HEMAOPHILIA THE ONLY CAUSE OF HEMATURIA? A SINGLE CENTRE EXPERIENCE FOR A DECADE

HemaSphere, 2023
Betül Kübra Tüzün, Zühal Demirci, Güray Saydam, Fahri Şahin   +3 more
doaj   +1 more source

Disease burden and unmet medical need in severe hemophilia in Greece: insights from clinicians and patients. [PDF]

Front Public Health
Sofiaki N, Zisimopoulou O, Solakidi A, Economou M, Katsarou O, Kouramba A, Nomikou E, Pergantou H, Vakalopoulou S, Filippidis G, Grammelis V, Menegas D, Angelopoulou A, Tzanetakos C, Gourzoulidis G.   +14 more
europepmc   +1 more source

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

Cardiac surgery in an infant hemophilia B carrier with moderate hemophilia: a case report. [PDF]

Front Cardiovasc Med
Hölz J, Nobile C, Holzapfel J, Lieftüchter V, De Winkel N, Olivieri M.   +5 more
europepmc   +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

Regional variations and trends in hemophilia prevalence: A global analysis with future projection. [PDF]

PLOS Glob Public Health
Mothashin M, Hossain MG, Barmon SC, Dipu MH, Saqlain MM, Mamun ASMA.   +5 more
europepmc   +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

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

, 1969
Brown, DW, Starzl, TE
core   +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

Optimizing Oligonucleotide Therapeutics: A Model‐Informed Drug Development Perspective

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Oligonucleotide therapies have emerged as a powerful therapeutic class, providing novel solutions for diverse diseases through antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers that specifically target and modulate gene expression or protein function. However, oligonucleotide development faces distinct challenges,
Ye Yuan, Vishnu Sharma, Venkatesh Atul Bhattaram, Xiaolei Pan, Justin Earp, Yun Wang, Jiang Liu, Hao Zhu   +7 more
wiley   +1 more source