Results 211 to 220 of about 1,419,402 (298)

Hypoxia and the cytoskeleton

The Journal of Physiology, EarlyView.
Abstract figure legend Schematic outlining the activation of hypoxia‐sensitive pathways, the influence of hypoxia and associated pathways on the cytoskeleton, and the impact of these on disease progression. Abstract A highly‐regulated and dynamic cytoskeleton is vital for functional cellular physiology and the maintenance of homeostasis.
Darragh Flood, Cormac T. Taylor
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

Clinical Outcomes of Efanesoctocog Alfa Prophylaxis in Children With Severe Haemophilia A: Interim Data From XTEND‐ed

Haemophilia, EarlyView.
Lynn Malec, Beatrice Nolan, Anthony K C Chan, Manuela Albisetti, Sheng‐Chieh Chou, Bulent Zulfikar, Mindy Simpson, Liqi Feng, Helena Palmborg, Graham Neill, Lydia Abad‐Franch, Sriya Gunawardena, Karin Fijnvandraat   +12 more
wiley   +1 more source

Physiological and anatomical determinants of placental drug transfer

The Journal of Physiology, EarlyView.
Abstract figure legend Fetal exposure to pharmaceuticals and their subsequent clearance back to the maternal circulation are governed by placental transfer. Passive diffusion down a maternal‐to‐fetal concentration gradient is the primary route of fetal drug exposure, with specific compounds undergoing transporter‐mediated transfer.
Rohan M. Lewis, Anandita Umapathy, Jane K. Cleal, Bram G. Sengers   +3 more
wiley   +1 more source

Drug Price and Health Policy Knowledge Influence Prescription Behavior in Orphan Diseases: Pheochromocytoma and Paraganglioma As Prototypes of Orphan Drug Econometrics. [PDF]

Cureus
Le DQ, Burton BN, Tejeda CJ, Hong JC, Lee JS, Kamdar N.   +5 more
europepmc   +1 more source

Sex‐Modulated TACSTD2 Methylation and Hormone‐Receptor Axis Interactions in Glioblastoma: Insights Toward Mechanistic Understanding

Computational and Systems Oncology, Volume 6, Issue 1, December 2026.
Sex‐stratified analyses of the TCGA and CGGA cohorts have identified promoter‐proximal TACSTD2 CpG sites where methylation shows opposite survival associations in males and females, is independent of copy number, and correlates with TACSTD2 expression.
Tahreem Fatima
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

The German ONKOPEDIA Guideline for Myelofibrosis in 2025—Recommendations of an MPN Expert Panel of the German Society for Hematology and Oncology (DGHO)

International Journal of Cancer, Volume 159, Issue 7, Page 1633-1641, 1 October 2026.
ABSTRACT The recently published ONKOPEDIA guideline on myelofibrosis, issued under the auspices of the German Society of Hematology and Oncology (DGHO), provides an updated, evidence‐based framework for the diagnosis and management of this rare, chronic myeloproliferative neoplasm.
Martin Griesshammer, Haifa Kathrin Al‐Ali, Gabriela M. Baerlocher, Konstanze Döhner, Steffen Koschmieder, Nicolaus Kröger, Petro E. Petrides, Dominik Wolf, Florian H. Heidel   +8 more
wiley   +1 more source

Advancing orphan drug development for rare diseases. [PDF]

Clin Exp Pediatr
Ko JM.
europepmc   +1 more source

Phenotypic expression of the gene‐for‐gene interaction in plant pathogenic bacteria: An implication for resistance to bacterial diseases by plants

Agrosystems, Geosciences &Environment, Volume 9, Issue 3, September 2026.
Abstract Plant pathogenic bacteria cause massive losses in agriculture and damage to natural plant habitats. Bacterial pathogens not only reduce crop production, but they also reduce crop quality by releasing toxins into the environment. Pathogenic bacteria that invade plant tissues must overcome the plant's defense mechanisms. Plants possess an innate
Muluken Getahun, Zelalem Bekeko
wiley   +1 more source