Caregivers and healthcare providers of patients with hemophilia both expressed unmet needs regarding information provision, a problem that may be remedied by using digital information platforms, ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Sithara Ramdas, MD, of Oxford Children’s Hospital in England, on neonatal and juvenile myasthenia gravis.

Sheila Jacobs is the president of STJ Editorial Services. For more than 25 years, Sheila has been providing writing, fact-checking, and editorial services to numerous clients in the medical education, ...

Women with pulmonary arterial hypertension (PAH) are 3 times more likely to have a history of adverse pregnancy outcomes (APOs) than women without PAH, according to results published in Pulmonary ...

Japanese women with Fabry disease experience “substantial emotional burdens” and do not receive support from their doctors and community, found a new study published in the scientific journal ...

A CRISPR/Cas9-modified Janus kinase 2 (JAK2) V617F model may be useful to develop new therapies against myeloproliferative neoplasms (MPNs), showed a new study published in the International Journal ...

Transthyretin amyloidosis (ATTR)-specific treatments and aortic valve replacement (AVR) are associated with significant survival benefits in patients with concomitant aortic stenosis and TTR amyloid ...

Physical activity before or during pregnancy was not associated with negative maternal/neonatal outcomes; however, physical activity was not statistically significantly associated with positive ...

The myocardium of patients with transthyretin amyloid cardiomyopathy (ATTR-CM) exhibits reduced contractile force compared to healthy cardiac muscle, according to a recent study published in JACC: ...

Capillary zone electrophoresis appears to effectively identify molecular fragments capable of binding to human transthyretin (TTR), with potential therapeutic applications for transthyretin amyloid ...

Among women who undergo routine obstetric antibody screening, certain maternal immune responses are occasionally detected that have an uncertain clinical significance. The current review focused on 4 ...

A recent meta-analysis published in Frontiers in Medicine has confirmed the clinical benefits of adeno-associated virus (AAV)-based liver-directed gene therapy for patients with hemophilia. “Our ...