One of the defining healthcare challenges of our age is the fair allocation of medical resources in the context of an ever-expanding population and runaway drug prices. The healthcare systems adopted ...

The new delivery system could effectively edit the huntingtin (HTT) gene in neurons derived from induced pluripotent stem cells obtained from patients with Huntington disease. A team of researchers ...

Multidisciplinary perisurgical care can best ensure the success of most surgeries for those with hemophilia. An 11-year-old boy presented with pain in his right thigh secondary to a fall 3 days prior.

Spatial transcriptomics and RNA sequencing identified upregulated cytokines and chemokines associated with lymphoid follicles and interfollicular regions in IgG4-RD. Spatial transcriptomics combined ...

Nipocalimab binds FcRn with high specificity, reducing IgG levels while sparing innate and adaptive immune functions. Nipocalimab effectively reduced maternal alloantibodies implicated in fetal and ...

BTKi monotherapy significantly prolonged time to next treatment or death compared with BCL-2i+CD20 monoclonal antibody in the second-line setting for CLL/SLL. Patients with chronic lymphocytic ...

An entrance to the CHEST 2024 conference in Boston, Massachusetts (Photo by Larry Luxner) The number of treatments for PAH continues to rise, but it still takes an an average of 3 years from symptom ...

Over 240 patients with ATTR-CM treated with tafamidis died over a median follow-up period of 43.2 months. More than a third (39%) of patients with transthyretin-mediated amyloid cardiomyopathy ...

In patients with hemophilia, the knee joints represent approximately 45% of all occurrences of joint bleeding. In patients with hemophilia, the effectiveness of knee replacement surgery is impacted by ...

Targeted disease-specific therapy should be considered in symptomatic patients with a confirmed diagnosis of post-orthotopic liver transplantation ATTR-CM. Many patients with hereditary ...

The lifetime risk of thrombosis in patients with PNH is approximately 50%, and it tends to be found in the abdominal or cerebral veins. Patients who experience thromboses at unusual sites should be ...

The evaluation of suspected cardiac amyloidosis should include screening tests for both AL and ATTR amyloidosis. A strong positive Tc-99 m pyrophosphate (PYP) scan, while generally indicative of ...