A KIT N822K mutation was found after progression in a boy with advanced systemic mastocytosis (SM) resistant to several lines ...

Lysosomotropic therapies such as hydroxychloroquine warrant caution in Fabry disease, as demonstrated in a case series.

Patients with AQP4-positive NMOSD who converted to seronegativity had a median annualized relapse rate of 0 in a Chinese ...

Patients with ITP treated with thrombopoietin receptor agonists as a second-line therapy had a longer time to next treatment ...

HSCT was linked to higher blood count normalization and failure-free survival rates than symptomatic treatment in patients ...

Forgetting to take medication represents the leading driver of suboptimal adherence to long-term prophylaxis in HAE, a study ...

According to a recent study, treatment trajectories for Wilson disease differ markedly across specialized German centers.

With only 3 HD therapies approved by the FDA, those with the disease are looking to new clinical trials for better answers ...

In a Huntington disease (HD) mouse model, an HSP90 inhibitor reduced mHTT aggregation and improved cognitive function and ...

Patients with progressive fibrosing interstitial lung diseases, including IPF and PPF, have to wait a long time to receive a ...

Virtual care for MG may alleviate physical and financial burdens, but its drawbacks could affect certain patient groups ...

Rh-null pregnancies can lead to a variety of clinical scenarios, including antenatal alloimmunization, HDFN, and severe fetal ...