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

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

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

There were fewer multiorgan transplants in patients with hereditary ATTR-CM with increased use of disease-modifying therapies, a study found.

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 ...

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 ...

A patient with massive hemoptysis was initially investigated for a lung tumor but was eventually diagnosed with IgG4-related respiratory disease. An elderly female patient who presented with recurrent ...

The analysis found no significant differences in estimated glomerular filtration rate changes or in the recovery of kidney function between the reduced-dose and standard-dose glucocorticoid groups. A ...

Liver stiffness measurement provided higher prognostic and diagnostic efficacy in low-risk patients compared with biochemical response criteria. Patients with primary biliary cholangitis (PBC) who ...

A patient with a working diagnosis of eosinophilic pneumonia was found to have EGPA upon further investigations. An elderly woman initially suspected of having eosinophilic pneumonia was eventually ...