Survey responses revealed a notable disconnect between how patients with PNH and their physicians perceive symptoms, ...
Analysis of a national registry of hospital data found racial gaps in blood transfusion rates among patients hospitalized for ...
The safety profile has generally been manageable, although cytopenias, gastrointestinal symptoms, and differentiation ...
Younger patients with MDS had lower-risk disease features and significantly better overall and leukemia-free survival.
A woman with hypoplastic low-risk MDS had reversible cytopenic flares in 2 pregnancies, with blood counts returning to normal ...
In a new study, nearly half of individuals with NF1 had cherry angiomas, suggesting a previously unrecognized link.
Mortality among older adults with MDS and CVD showed substantial disparities by age, sex, race, region, and urbanization.
Older adults with primary glomerular diseases (PDGs) like FSGS had greater comorbidity and renal chronicity but similar ...
UniQure advanced ifezuntirgene inilparvovec (AMT-130), a gene therapy for Huntington disease (HD), toward potential US and UK ...
Clinical trials of 2 CAR T-cell therapies in MG, MS, and a range of autoimmune diseases have been paused following safety ...
A woman living with HAE recalls her worst swelling episode yet and how it has affected the way she views the good days.
Premature consolidation affected 8.9% of lengthened segments in children with achondroplasia, with the fibula most frequently ...
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