Italian researchers found that 62% of patients with early-stage Huntington disease (HD) lacked insight into their motor ...
The US Food and Drug Administration (FDA) has accepted a supplemental New Drug Application for nebulized treprostinil for the ...
The safety profile has generally been manageable, although cytopenias, gastrointestinal symptoms, and differentiation ...
In a new study, nearly half of individuals with NF1 had cherry angiomas, suggesting a previously unrecognized link.
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 ...
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 ...
A woman living with HAE recalls her worst swelling episode yet and how it has affected the way she views the good days.
A woman with CIDP explains what it feels like when doctors test how quickly electrical pulses can travel through her ...
Findings of a new study suggest the 12-minute walk test is of limited value in the diagnosis of mitochondrial myopathies such ...
Premature consolidation affected 8.9% of lengthened segments in children with achondroplasia, with the fibula most frequently ...