Updated guidelines for managing patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) from the British Society for Rheumatology (BSR) were published recently in ...

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.

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 ...

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 ...

Models of trajectories for disease progression and placebo response were developed for patients with Huntington disease (HD) using natural history data from the Enroll-HD clinical trial and the ...

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 ...

VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly identified disease that can manifest as vasculitis (commonly, small-vessel vasculitis), making it a challenge to ...

Treatment with porcine antihuman T-lymphocyte globulin (ATG) and cyclosporine, with or without a thrombopoietin receptor agonist (TPO-RA), was effective and generally well tolerated as first-line ...

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 ...

Researchers from Iran presented the case of a patient with chronic inflammatory demyelinating polyneuropathy (CIDP) who underwent emergency umbilical herniorrhaphy and whose anesthesia was ...