In a recent study, a real-world cohort of patients with ATTR-CM treated with tafamidis demonstrated improved survival ...

Calcineurin inhibitor-associated injury accompanied de novo FSGS in nearly half of affected allograft biopsies.

Pulmonary hypertension is common among patients with PPF, and the rates are comparable to those of patients with IPF, ...

A woman with FA tries 2 types of CPAPs to treat her sleep apnea. One worked better, but she’s still sleepless. Here’s why.

Positive levels of circulating anti-nephrin autoantibodies increase the risk of FSGS recurrence after kidney transplant.

MCL therapy is shifting toward targeted and immune-based approaches, making the order of treatment increasingly consequential ...

Dual reconstruction plates accommodated the shortened, angulated anatomy of an achondroplastic distal humerus.

Immunosenescence offers a novel framework for IPF pathogenesis and treatment, though associated biomarkers and therapies ...

In cellular models, curcumin affected several HD-related pathways, suggesting it may be useful as a tool for studying disease ...

A report of 2 patients Dravet syndrome (DS) who developed ARDS suggests it may be an underrecognized extraneurologic feature ...

A sports coach and athlete explains how living with Pompe disease has changed his perspective on the purpose of exercising.

In a real-world study, nearly half of adults with LGS, DS, or TSC who added CBD experienced a 50% or greater reduction in ...