Results 111 to 120 of about 13,095,395 (361)
Pediatric Blood &Cancer, EarlyView.ABSTRACT Objectives The association between exposure to dinutuximab beta (DB) and event‐free survival (EFS) or overall survival (OS) of neuroblastoma patients was assessed using data collected during three clinical trials (five cohorts). Methods A systematic review (March 2026) was conducted to identify relevant studies (prospective; registered DB ...
Przemysław Holko +19 more
wiley +1 more source
The Challenge of Rare Diseases [PDF]
Chest, 2018 Rare diseases pose particular challenges to patients who are affected, to the clinicians who care for them, and to the investigators who study their conditions. Although individually uncommon, rare diseases are common in the aggregate, with approximately 7,000 described rare diseases affecting 25 to 30 million US adults.
openaire +2 more sources
Orphanet Journal of Rare Diseases, 2021 Background There are more than 7000 rare diseases, most of which have no specific treatment. Disease profiles, such as prevalence and natural history, among the population of a specific country are essential in determining for which disease to research ...
Kota Ninomiya, M. Okura
semanticscholar +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Hemophilic arthropathy remains the leading morbidity in hemophilia despite modern prophylaxis, and early joint damage may be missed by routine exams. This study explored T2* MRI as a noninvasive biomarker of hemosiderin deposition in pediatric hemophilia.
Jessica Garcia +6 more
wiley +1 more source
Study on Drug List and Accessibility of Rare Diseases Based on the China′s Second List of Rare Diseases [PDF]
Objective This study aims to sort out the rare disease drugs in the China′s Second List of Rare Diseases, to provide reference for the management of rare disease drug treatment.
ZHANG Bo +5 more
core +1 more source
Studying Epidemiological Properties Of Rare Diseases [PDF]
, 2021 Introduction: A type of disease where a small percentage of the population has been affected are called rare diseases. The rare diseases subject is full of misunderstandings.
Alam, Jamal
core
Collaborative model for diagnosis and treatment of very rare diseases: experience in Spain with thymidine kinase 2 deficiency [PDF]
, 2021 Malaltia mitocondrial; Medicina mitocondrial; Deficiència de timidina cinasa 2 (TK2d)Mitochondrial disease; Mitochondrial medicine; Thymidine kinase 2 deficiency (TK2d)Enfermedad mitocondrial; Medicina mitocondrial; Deficiencia de timidina cinasa 2 (TK2d)
Domínguez-Gonzalez, C. +6 more
core +1 more source
Exploring ITM2A as a new potential target for brain delivery
Fluids and Barriers of the CNS, 2022 Background Integral membrane protein 2A (ITM2A) is a transmembrane protein expressed in a variety of tissues; little is known about its function, particularly in the brain.
Céline Cegarra +9 more
doaj +1 more source
Collecting rare diseases [PDF]
F1000Research, 2014 This editorial introduces the F1000Research rare disease collection. It is common knowledge that for new treatments to be successful there has to be a partnership between the many interested parties such as the patient, advocate, disease foundations, the academic scientists, venture funding organizations, biotech companies, pharmaceutical companies ...
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Pediatric Blood &Cancer, EarlyView.ABSTRACT Purpose Despite 5‐year survival rates of over 90% among children and adolescents/young adults (CAYAs) with classic Hodgkin lymphoma (cHL), 15%–20% relapse after frontline therapy. Prior analysis of frontline Children's Oncology Group (COG) clinical trials demonstrated that, despite similar rates of relapse, non‐Hispanic Black (NHB) and ...
Mallorie B. Heneghan +14 more
wiley +1 more source