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

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

ATTR-PN diagnosis requires a combination of neurological and non-neurological tests, such as blood pressure monitoring, a ...

A recent analysis of the PEXIVAS trial showed that plasma exchange can significantly improve kidney function in patients with AAV and kidney involvement.

Recent findings indicate that the new BTK inhibitors, although not targeting TEC kinases, may be responsible for fungal infections via a disruption of the signal downstream of BTK, responsible for ...

Test Your Knowledge About the Diagnosis of Alpha-1 Antitrypsin Deficiency Test Your Knowledge About the Symptoms of Patients With AATD Test Your Knowledge About the Prognosis of Patients With AATD ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

High incidence, morbidity, and mortality rates due to hemolytic disease of the fetus and newborn (HDFN) have been observed in Latin America.

The potential of axonal damage as a biomarker of disease severity and predictor of further outcomes is highlighted by its effect on functional disability, even in early disease stages. Patients who ...

The Chasing Rainbows Foundation raises awareness of rare pregnancy-related diseases. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Erin Collins, founder of the Chasing ...