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 ...

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

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

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

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

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

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

Missing EEG findings and inconsistent documentation make it difficult for researchers to identify LGS from electronic health ...

A woman with Friedreich ataxia (FA) says living with her rare disease is a balancing act that requires tremendous patience.

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

A recent study demonstrated iptacopan added to rituximab and a glucocorticoid taper may reduce proteinuria earlier than ...