Results 161 to 170 of about 32,298 (248)

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

Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries. [PDF]

Front Pharmacol
Jakubowski S, Holko P, Nowak R, Warmuth M, Dooms M, Salminen O, Cortial L, Selke GW, Georgi C, Magnússon E, Crisafulli S, Strijbosch F, Mueller T, Grieve E, Danés I, Kawalec P.   +15 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

Orphan drugs: The final frontier in the fight against rare diseases.

Indian J Pharmacol
Rajaram S, Medhi B.
europepmc   +1 more source

Results of health technology assessments of orphan drugs in Germany-lack of added benefit, evidence gaps, and persisting unmet medical needs. [PDF]

Int J Technol Assess Health Care
Kranz P, McGauran N, Ünal C, Kaiser T.
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

Implementation status of pharmacological studies in the development of orphan drugs. [PDF]

Orphanet J Rare Dis
Yokoshiki S, Arato T.
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

Characterization of Adult Patients With Neurometabolic Disorders: A Cross‐Sectional Study at a Tertiary Neurology Center in Sweden

JIMD Reports, Volume 67, Issue 5, September 2026.
ABSTRACT Adult patients with inherited metabolic diseases are often overlooked. Limited data on this population hinder adequate planning of their clinical and social care. In this retrospective, observational, cross‐sectional service evaluation study, we reviewed the electronic medical records of adult patients with inherited neurometabolic diseases ...
Boel Ernerdahl, Ashraf Yahia, Andreas Puschmann   +2 more
wiley   +1 more source