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 ...

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 ...

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.

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 ...

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 ...

The present study did not find genetic evidence of a causal association between PBC and pregnancy outcomes, which is consistent with the findings of several previous studies. Genetically predicted ...

The use of short femoral stems may offer a suitable surgical strategy for addressing the complex femoral anatomy associated with achondroplasia. Total hip arthroplasty (THA) in individuals with ...

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 ...

The new mouse line was treated with avapritinib, a tyrosine kinase inhibitor that is approved for the treatment of SM in humans. A new inducible transgenic mouse model has been developed that has the ...

Friedreich ataxia (FA) is a constant challenge. I know that. My loved ones know that. My doctors know that too. But many others realize the extent of what it’s like beyond the surface. In college, I ...