Results 71 to 80 of about 2,408,361 (302)

Organoids in pediatric cancer research

FEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source

Rare diseases [PDF]

Thorax, 1999
C M ROBERTS;, A E TATTERSFIELD, R M DU BOIS   +2 more
openaire   +4 more sources

PLASMIC score to aid diagnosis of aHUS: an analysis of C5 inhibitor clinical trials and the PINC AI™ healthcare database

BMC Nephrology
Background Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) that can lead to end organ damage and death without treatment.
Miguel G. Uriol-Rivera, Frank R. Ernst, John N. Booth, Àngels Comas, Christoph Gasteyger, Ioannis Tomazos, Ching Lum, Yan Wang, Ana I. Ávila   +8 more
doaj   +1 more source

Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs [PDF]

, 2018
While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment.
Bannister, Julien B.
core   +1 more source

Structural biology of ferritin nanocages

FEBS Letters, EarlyView.
Ferritin is a conserved iron‐storage protein that sequesters iron as a ferric mineral core within a nanocage, protecting cells from oxidative damage and maintaining iron homeostasis. This review discusses ferritin biology, structure, and function, and highlights recent cryo‐EM studies revealing mechanisms of ferritinophagy, cellular iron uptake, and ...
Eloise Mastrangelo, Flavio Di Pisa
wiley   +1 more source

Population PK–PD Modeling of Danicopan Add‐On Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria Treated With Ravulizumab or Eculizumab

CPT: Pharmacometrics & Systems Pharmacology
Danicopan is a first‐in‐class orally administered complement factor D inhibitor, approved as an add‐on therapy to ravulizumab or eculizumab for the treatment of clinically significant extravascular hemolysis in patients with paroxysmal nocturnal ...
Jun Chen, Feng Yang, Hanbin Li, Rajendra Pradhan, Stephan Ortiz   +4 more
doaj   +1 more source

A Comprehensive Study of the Effects by Sequence Truncation within Inverted Terminal Repeats (ITRs) on the Productivity, Genome Packaging, and Potency of AAV Vectors

Microorganisms
One of the primary challenges in working with adeno-associated virus (AAV) lies in the inherent instability of its inverted terminal repeats (ITRs), which play vital roles in AAV replication, encapsidation, and genome integration.
Yinxing Chen, Shiliang Hu, William Lee, Noel Walsh, Kayla Iozza, Neil Huang, Gregory Preston, Lauren M. Drouin, Nannan Jia, June Deng, Matthias Hebben, Jing Liao   +11 more
doaj   +1 more source

Disseminated cutaneous gout: a rare manifestation of a common disease [PDF]

, 2020
Disseminated cutaneous gout is a rare atypical cutaneous manifestation of gout in which widespread dermal and subcutaneous tophi develop at extra-articular body sites.
Adler, Brandon L, Crew, Ashley, DeClerck, Brittney, Guzman, Ruben, Peng, David   +4 more
core  

Organ‐specific redox imbalances in spinal muscular atrophy mice are partially rescued by SMN antisense oligonucleotides

FEBS Letters, EarlyView.
We identified a systemic, progressive loss of protein S‐glutathionylation—detected by nonreducing western blotting—alongside dysregulation of glutathione‐cycle enzymes in both neuronal and peripheral tissues of Taiwanese SMA mice. These alterations were partially rescued by SMN antisense oligonucleotide therapy, revealing persistent redox imbalance as ...
Sofia Vrettou, Brunhilde Wirth
wiley   +1 more source

Establishment of an α-thalassemia mouse model through fetal liver cell transplantation and analysis of hematological parameters

Scientific Reports
Clinically, α-thalassemia is stratified into mild, intermediate, and severe forms, differentiated by the degree of anemia severity. Specifically, severe α-thalassemia manifests in homozygous individuals, characterized by a profound α globin deficit.
Xin Xu, Wencheng Fu, Wenrui Ye
doaj   +1 more source