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

Routine immunoglobulin G (IgG) monitoring may help identify pediatric patients with acute lymphoblastic leukemia (ALL) at risk for hypogammaglobulinemia and frequent febrile episodes during ...

Nipocalimab is a fully human, IgG1, high-affinity monoclonal antibody that blocks placental IgG transfer and reduces levels of circulating maternal IgG.

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.

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

Özge’s background is in research; she holds a MSc. in Molecular Genetics from the University of Leicester and a PhD. in Developmental Biology from the University of London. Özge worked as a bench ...

Gaucher disease is caused by a mutation in the GBA1 gene, which encodes the enzyme lysosomal acid glucosylceramidase. This enzyme breaks down glucocerebroside to form glucose and ceramide within ...

Perampanel may be safe and effective as a second- or third-line adjunctive therapy for the treatment of Lennox-Gastaut syndrome (LGS), a study found.

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.