Researchers found that pediatric male patients with hemophilia are at a greater risk of falls due to poor dynamic balance.

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

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 ...

Alpha-1 antitrypsin deficiency (AATD) is found to be associated with pregnancy-related complications and adverse birth ...

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) ...

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.

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...