The 3 patients who underwent surgical gallstone removal all ultimately recovered well, including one who developed acute chest syndrome. As cholelithiasis and choledocholithiasis are common in ...

The targeting of factor XII activation is a key strategy in offering long-term prophylaxis to patients with hereditary angioedema. Garadacimab, a therapeutic targeting factor XII activation, has been ...

Pretreating human astrocytes with interleukin (IL)-17A, IL-10, and IL-6 could help differentiate between serum obtained from anti-aquaporin 4 (AQP4) antibody-positive NMOSD and healthy controls.

Duchenne muscular dystrophy (DMD) is a progressive and fatal genetic disease of skeletal muscle wasting, primarily affecting boys in early childhood. Traditional therapies, such as systemic ...

Recent research has shown that intestinal barrier disruption worsens experimental AIP by facilitating the translocation of pathogenic bacteria, including Staphylococcus sciuri, from the gut to the ...

Patients with GPP had high treatment persistence on ixekizumab, with significant PASI score improvements. Ixekizumab maintained a favorable safety and efficacy profile across psoriasis subtypes, ...

A phase 3 study is ongoing to confirm the survival benefit of venetoclax plus azacitidine in patients with treatment-naive high-risk MDS. The combination of venetoclax plus azacitidine was well ...

Hemophagocytic lymphohistiocytosis, a severe hyperinflammatory condition, can complicate FNAIT, making early diagnosis critical. Hemophagocytic lymphohistiocytosis (HLH), a life-threatening ...

Collaboration between clinicians and caregivers is a key component to supporting effective use of the CGI-I Non-seizure Symptoms measure. The CGI-I Nonseizure Symptoms measure was developed to be ...

This is the first reported case of a child with PWS who was further diagnosed with LGS. “To our knowledge, this is the first reported case of LGS in a pediatric patient with Prader-Willi syndrome in ...

Having RA and type 1 diabetes increased the risk of developing PAH, and a link was found between a genetic risk of PAH and an increased risk of PAH. Bidirectional Mendelian randomization (MR) analysis ...

Art Still is a former defensive end for the Kansas City Chiefs and an NFL Hall of Famer. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Art Still, former defensive end for the ...