Modern prophylactic therapies for hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) have become increasingly prevalent in Hungary, paralleling global trends, and are proving both ...

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 ...

To assess the effect of NMOSD, MS, and related diseases on employment and income, a team of researchers, led by Jiraporn Jitprapaikulsan, MD, from the Mahidol University in Bangkok, Thailand, ...

When comparing the digital and paper formats of the eMPhasis-10, the responses from patients showed remarkable consistency. In other words, there was a strong association between both digital and ...

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 ...

Vosoritide, a once-daily injectable therapy, may improve growth outcomes in children with achondroplasia when administered continuously from infancy to final adult height, according to findings of a ...

No new cases of Fabry disease have been identified in 8 renal centers in the UK, in spite of the implementation of stringent screening criteria, according to a new study published in the journal BMC ...

Researchers from Brazil developed a new clinical score to estimate the probability of neuromyelitis optica spectrum disorder (NMOSD) in patients presenting with optic neuritis. The easily accessible ...

Clinically relevant concentrations of deoxycytidine (dC) and deoxythymidine (dT) are unlikely to cause or be subject to pharmacokinetic drug-drug interactions, according to a new study published in ...

In patients with hepatocellular carcinoma (HCC), serum alpha-fetoprotein (AFP) levels are unreliable as a sole biomarker for disease diagnosis, according to findings from a retrospective study ...

Individuals with homozygous ZZ alpha-1 antitrypsin deficiency (AATD) face a substantial burden of liver disease, according to results of a meta-analysis recently published in Liver International ...

Two phase 3 clinical trials, MOBILIZE and VITALIZE, are recruiting patients with chronic demyelinating polyneuropathy (CIDP) to evaluate the effectiveness of riliprubart according to an abstract ...