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, ...

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, ...

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 ...

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.

Gamunex ® -C (immune globulin injection [human]) is a specific intravenous immunoglobulin (IVIg) indicated for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in adults to ...

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 ...

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 ...

“GH replacement has been approved in Japan since 2002 for ‘short stature without epiphyseal closure associated with PWS,’ but at the time the study was conducted, it was not yet approved for improving ...

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 ...

SM diagnosis analysis between 2000 and 2021 revealed an incidence of 0.1 per 100,000 in non-Hispanic whites compared with 0.04 per 100,000 among Hispanics.

Which of the statements below about the epidemiology of generalized pustular psoriasis (GPP) are correct? Select one of the options for each question, then submit your answers to see how well you ...