Results 61 to 70 of about 7,998,845 (308)
ABSTRACT Background Graft‐versus‐host disease (GvHD) remains one of the most significant complications of hematopoietic stem cell transplantation (HSCT). Abatacept, a selective T‐cell costimulation modulator, is a relatively novel prophylactic agent, though pediatric data remain limited.
Matthijs D. Kruizinga +3 more
wiley +1 more source
Summary of Research: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis
This article provides a summary of a previously published paper: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. The paper reported the results of the CHAMPION-MG trial which investigated the drug ravulizumab in the rare ...
Tuan Vu +9 more
doaj +1 more source
ABSTRACT Background and Aims Infant bone and soft tissue neoplasms present distinct clinical challenges and lack large‐scale data to inform treatment decisions. This study aims to characterize bone and soft tissue neoplasms in infants in Canada and to compare their outcomes with those of older pediatric patients.
Samuel Sassine +21 more
wiley +1 more source
Introduction Neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease, affects the optic nerves and spinal cord. Rituximab is often used off-label to treat NMOSD; however, comparator-controlled trial data are limited.
Justin Lee +4 more
doaj +1 more source
ABSTRACT Background Outcomes in relapsed or refractory pediatric and AYA bone sarcomas remain poor, and the impact of early referral to specialized sarcoma centers on survival is unclear. Early centralization of care may improve local and systemic treatment quality, but evidence in this population is limited.
Justyna Antoniuk‐Majchrzak +9 more
wiley +1 more source
Expert Consensus for the Diagnosis and Treatment of Bartter Syndrome in China(2023)
Bartter syndrome (BS) is a rare inherited salt-losing renal tubular disorder characterized by secondary hyperaldosteronism with hypokalemia and hypochloremic metabolic alkalosis, and normal or low blood pressure.
Chinese Society of Rare Diseases +4 more
doaj +1 more source
Performance of genotype imputation for rare variants identified in exons and flanking regions of genes. [PDF]
Genotype imputation has the potential to assess human genetic variation at a lower cost than assaying the variants using laboratory techniques. The performance of imputation for rare variants has not been comprehensively studied.
Margaret Gelder Ehm +50 more
core +2 more sources
ABSTRACT Background Therapeutic apheresis (TA) is an established treatment modality for hematologic, neurologic, and immunologic disorders, yet access remains severely limited in sub‐Saharan Africa. Donor apheresis, including platelet apheresis collection from healthy donors, represents an important complementary modality supporting blood product ...
Nosa Bazuaye +33 more
wiley +1 more source
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 +8 more
doaj +1 more source
A rare presentation of a rare disease [PDF]
A 29 year old woman presented to the gynaecology services with a history of cervical smear cytology and punch biopsy showing human papillomavirus related changes, associated with moderate dyskaryosis. She had undergone laser loop excision biopsy of the transformation zone (LLETZ) two years before for previous abnormal smears.
Y Steppeler, H Shaikh
openaire +1 more source

