Careful attention to blood sample handling, including maintaining a warm chain, can help identify cold agglutinin disease (CAD).

Serum transthyretin levels appear to correlate with worse prognosis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a study found.

The suspicion of cardiac amyloidosis is still age-biased among healthcare professionals. In spite of recent educational efforts.

KIT inhibitors such as midostaurin may offer meaningful clinical benefit in patients with aggressive systemic mastocytosis.

Patisiran was associated with a moderate improvement in cardiac symptoms in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a study found.

The US Food and Drug Administration (FDA) notified Sarepta Therapeutics that it may now lift the ban on the shipment of its Duchenne muscular dystrophy (DMD) drug, delandistrogene moxeparvovec ...

Using the new method, the researchers were able to successfully identify 15 cases of transthyretin mediated amyloid cardiomyopathy (ATTR-CM).

Results of the ApproaCH trial showed that navepegritide improved AGV over placebo in pediatric patients with achondroplasia.

Models of clinical score trajectories were developed using natural history data from patients with HD and tested against placebo response.

A patient with DADS deteriorated and presented with CIDP-like symptoms after treatment with rituximab, according to case report.

Subcutaneous immunoglobulin delivered via prefilled syringes and manual push is well-tolerated in patients with CIDP, a study found.

Novel findings in the pathophysiology of HD could unlock a new imaging-based marker for disease burden and clinical decline, a study found.