Results 41 to 50 of about 3,489,004 (303)
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
Scientific ReportsBIVV003 is a gene-edited autologous cell therapy in clinical development for the potential treatment of sickle cell disease (SCD). Hematopoietic stem cells (HSC) are genetically modified with mRNA encoding zinc finger nucleases (ZFN) that target and ...
Samuel Lessard +27 more
doaj +1 more source
Sleep Disorders in Childhood Neurogenetic Disorders
, 2017 Genetic advances in the past three decades have transformed our understanding and treatment of many human diseases including neurogenetic disorders. Most neurogenetic disorders can be classified as “rare disease,” but collectively neurogenetic disorders ...
Laura Dosier +5 more
core +1 more source
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
An algorithm as a diagnostic tool for central ocular motor disorders, also to diagnose rare disorders. [PDF]
, 2019 BACKGROUND Recently an increasing number of digital tools to aid clinical work have been published. This study's aim was to create an algorithm which can assist physicians as a "digital expert" with the differential diagnosis of central ocular motor ...
Kremmyda, Olympia +5 more
core +3 more sources
, 2014 The European Union has funded the development of a new disease registry for McArdle disease and other rare glyco (geno) lytic disorders presenting with exercise intolerance.
Baruch, N. +39 more
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Pediatric intramedullary low‐grade gliomas (PIMLGGs) are rare neoplasms that present unique clinical and management challenges. Although surgery remains the primary tool for tissue diagnosis and decompression, complete gross‐total resection is frequently unfeasible due to the infiltrative nature of these lesions.
Olga M. Sergeenko +5 more
wiley +1 more source
Orphanet Journal of Rare Diseases, 2022 Background Experienced fatigue is an under-recognized and under-researched feature in persons with many different rare diseases. A better overview of the characteristics of existing research on experienced fatigue in children and adults with rare ...
Trine Bathen +3 more
doaj +1 more source
Undiagnosed Rare Genetic Disorders: Importance of Functional Characterization of Variants
, 2023 Rare Genetic Disorders (RGDs) are defined as disorders that affect less than 1 in 2000 people, and collectively affect more than 300 million people worldwide [...
Muhammad Umair, Ahmed Waqas
core +1 more source
Pediatric Blood &Cancer, EarlyView.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