Patients with Wilson disease are at risk of osteoporosis and osteopenia, particularly older, male patients and those with skeletal symptoms.

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

Autoimmune neonatal thrombocytopenia may be the first clinical indication of undiagnosed maternal systemic lupus erythematosus.

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.

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.

The presence of intracutaneous amyloid deposition in asymptomatic TTR mutation carriers could be a predictive of ATTR-CM progression.

A new set of consensus-based statements (CSs) on appropriate myelofibrosis (MF) management for the Asia-Pacific (APAC) region.

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

Acquired resistance to venetoclax in chronic lymphocytic leukemia (CLL) was strongly associated with the development of complex karyotypes, particularly those marked by unbalanced translocations on ...