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.

Human urine-derived renal epithelial cells replicate key structural and molecular markers of Fabry disease, a study found.

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

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 ...

Tafamidis is linked to reduced all-cause mortality and a lower incidence of congestive heart failure exacerbations in ATTR-CM, a study found.

A multimodal approach combining vosoritide and limb lengthening led to gains in stature, gait, and emotional well-being in achondroplasia.

Neonatal anthropometric measurements may serve as indicators for the early detection of skeletal dysplasias, including achondroplasia.

Conducting a RCT of the Accessercise app among adults with physical disabilities, including achondroplasia, is feasible and acceptable.

Are you a health care provider looking for information on the life expectancy of Fabry disease? Read more about it here.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...