Fingolimod treatment leads to beneficial effects in Huntington disease (HD), according to a new study in a mouse model of the ...

The dried blood spot test approach requires no special handling or storage and can be carried out with capillary instead of venous blood. A new study has found that a novel functional C1-INH (fC1-INH) ...

Artificial intelligence may improve the diagnostic rates of AAV in a cost-effective manner. Deep learning models may be useful in identifying potential cases of antineutrophil cytoplasmic antibody ...

The researchers used latent class analysis with 10 indicator variables such as ANCA subtype and tracheal and bronchial involvement to identify subtypes of AAV. A new study has employed latent class ...

Which of the following statements about rare diseases and their treatments are correct? Select one of the options for each question, then submit your answers to see how well you scored and how other ...

If successful, NTLA-2002 could significantly alleviate treatment burdens while improving the quality of life for individuals affected by this debilitating condition. The phase 3 HAELO trial for ...

These findings may help to inform future treatment discussions and enhance aspects of physician-patient dialogue for patients on long-term prophylaxis and those using on-demand therapy only.

Multiple steps are involved in the pathogenesis of chronic lymphocytic leukemia (CLL) and various factors are related to its development: cytogenetic aberrations, antigenic triggers activated by ...

This is a picture of my dad, and it fills me with many emotions: sadness, grief, gladness, and relief. This is the picture I used on my blog to share my journey with transthyretin-mediated amyloid ...

In patients with In Becker muscular dystrophy BMD) and myotonic dystrophy type 1, the amplitude ratio of the movement-related potential did not change significantly when the duration of the ...

All patients presented to the hospital with acute myelitis in the form of weakness in the arms and legs but no concurrent visual complaints. A team of researchers from Ethiopia presented a case series ...

All 5 patients who switched from eculizumab remained relapse-free while being treated with satralizumab. Satralizumab after eculizumab is effective and well tolerated in patients with neuromyelitis ...