Results 41 to 50 of about 4,732,768 (313)
Orphanet Journal of Rare Diseases, 2021 Background One objective of the Belgian Rare Diseases plan is to improve patients’ management using phenotypic tests and, more specifically, the access to those tests by identifying the biochemical analyses used for rare diseases, developing new ...
Nathalie M. Vandevelde +20 more
doaj +1 more source
Expert Guidelines for the Diagnosis, Treatment, and Management of Mycosis Fungoides in China
罕见病研究, 2023 Mycosis fungoides (MF) is a cutaneous lymphoma originating from memory helper T cells. The lesion caused by classical type of MF is characterized by the progression from patches at early stages, advancing to more infiltrated plaques and eventually to ...
Rare Skin Disease Committee of China Alliance for Rare Diseases +2 more
doaj +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Rates of cancer among adolescents and young adults (AYA), age 15–39 years, are increasing. Consequently, radiation oncologists are treating more AYAs who have diagnoses spanning both pediatric and adult practices. Compared to pediatric and older adult patients, AYAs face a unique set of challenges.
Hesham Elhalawani +7 more
wiley +1 more source
A brief insight into the rare diseases in Egypt
, 2023 Rare diseases (RDs) are a group of lifetime incapacitating or fatal diseases affecting nearly 3.5–5.9% of the global population, reaching 263–446 million individuals.
Gehad Atef Oura +7 more
core +1 more source
National programme for rare diseases 2019–2023
, 2020 It is estimated that six out of one hundred Finns have a rare disease, injury, syndrome or malformation. It can sometimes be challenging and time-consuming to identify them.
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Allogeneic hematopoietic stem cell transplantation (alloHSCT) is an essential therapy for several malignant and nonmalignant diseases, but relapse and graft loss remain the principal threats to its success. Routine monitoring of chimerism and minimal residual disease (MRD) enables early detection of imminent recurrence and guides ...
Carmen Junk +10 more
wiley +1 more source
The Diagnosis and Treatment of Rare Diseases in Children: Now and Future
, 2022 Rare diseases refer to a group of diseases having very low incident rates in the population without unified definition up till now. Approximately 50% to 75% of rare diseases occur at birth or in childhood, incurring huge psychological and economic burden
NI Xin, HAO Chanjuan
core +1 more source
Consensus of Chinese Experts on the Diagnosis and Treatment of Type Ⅰ Interferonopathy
罕见病研究Type Ⅰ interferonopathy is an autoinflammatory disease that affects multiple systems, with a high disability and mortality rate. It profoundly impacts the quality of life of patients and poses a considerable burden on their families and society.
Chinese Alliance of Pediatric Rheumatic & Immunologic Diseases +1 more
doaj +1 more source
Journal of Investigative Medicine High Impact Case Reports, 2016 Erdheim-Chester disease (ECD) is a rare, xanthogranulomatous, non-Langerhans cell histiocytosis with frequent systemic involvement. Although the diagnosis is based on characteristic histological and radiological findings, its identification can be challenging because of its heterogeneous presentation. Osteosclerosis of long bones, often associated with
Matzumura, Melissa +2 more
openaire +2 more sources
Pediatric Idiopathic Multicentric Castleman Disease Is Often Severe But Responsive to Siltuximab
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Idiopathic multicentric Castleman disease (iMCD) is a potentially fatal immunologic disorder marked by widespread lymphadenopathy and inflammation. Siltuximab, an interleukin‐6 (IL‐6) inhibitor, is the only FDA‐approved treatment for adult patients with iMCD.
Bridget Austin +17 more
wiley +1 more source