The updated 2025 BSR management recommendations for patients with AAV provide systematic and evidence-based recommendations to support UK clinicians in managing AAV across the entire life course.

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.

ATTR treatments and aortic valve replacement are associated with significant survival benefits in patients with aortic stenosis and ATTR-CM.

Vosoritide can improve growth outcomes in children with achondroplasia when administered continuously from infancy to final adult height.

Researchers developed a new clinical score to estimate the probability of NMOSD in patients presenting with optic neuritis.

Clinically relevant concentrations of dC and dT are unlikely to cause or be subject to pharmacokinetic drug-drug interactions, a study found.

Models of clinical score trajectories were developed using natural history data from patients with HD and tested against placebo response.

Individuals with homozygous ZZ alpha-1 antitrypsin deficiency (AATD) face a substantial burden of liver disease.

The close similarities between VEXAS syndrome and primary vasculitis disorders make them hard to distinguish clinically.

In elderly patients with severe aplastic anemia (SAA), immunosuppressive therapy with or without TPO-RA was effective and safe.

Two phase 3 clinical trials are recruiting patients with CIDP to evaluate the efficacy of riliprubart treatment.

Researchers presented the case of a patient with CIDP who had emergency umbilical herniorrhaphy and whose anesthesia was successfully managed.