The discovery of biomarkers associated with inhibitor development may boost preventative treatment strategies. Raised levels of granulocyte colony-stimulating factor (G-CSF) and interleukin-6 (IL-6), ...

Studies demonstrate that the burden of an achondroplasia diagnosis is high, taking a toll on both the patient and their family. Achondroplasia is an incurable, lifelong condition that is primarily ...

Which of the statements below about experimental therapies for antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) are correct? Select one of the options for each question, then ...

A substantial proportion of caregivers reported improvements in a wide range of non-seizure and seizure outcomes. CBC has recognized efficacy in reducing seizure frequency in LGS and DS; however, its ...

Combined use of CE-T1 and T2 MRI sequences showed significant advantages over using a single MRI sequence in classifying and identifying CNS tumors. A new magnetic resonance imaging (MRI)-based ...

Patients with NMOSD demonstrated significantly higher adrenergic dysfunction compared to patients with MS. In addition, the cardiovascular autonomic dysfunction scores of patients with NMOSD were also ...

In HD, striato-cortical circuits appear to engage in cross-talk and reorganization, potentially serving compensatory mechanisms in response to neurodegeneration. Distinct clinical profiles of ...

The current estimates suggest around 500 to 600 patients having transthyretin amyloidosis (ATTR) in France but from 2018 and 2023 a total of 1,179 people were identified as having a pathogenic TTR ...

Hematomas obstructing the airway are a potentially life-threatening complication in severe hemophilia. A young man with severe hemophilia A had a pharyngolaryngeal hematoma that could potentially ...

Approximately one-third of survey participants had never been treated with pirfenidone or nintedanib. People living with idiopathic pulmonary fibrosis (IPF) face significant challenges, including gaps ...

Patients with primary myelofibrosis (MF) have the highest risk of pulmonary hypertension among patients with Philadelphia chromosome-negative myeloproliferative neoplasms (Ph-MPNs), found a new study ...

Most patients with LR-MDS do not achieve durable RBC-TI with currently available therapies, highlighting a clear unmet clinical need. In patients with lower-risk myelodysplastic syndromes (LR-MDS), ...