A recent analysis of 2,597 responses to clinical quizzes highlights the educational impact of the AA Challenge program on understanding AA amyloidosis. This interactive French-language initiative aims to enhance knowledge about the causes of this rare disease.

A retrospective study highlights the clinical burden of AA amyloidosis in four historical monogenic autoinflammatory diseases. Insights from this research may inform future therapeutic strategies and patient management.

A case report highlights the occurrence of AA amyloidosis with multiorgan involvement as a complication of severe hidradenitis suppurativa. This finding underscores the need for awareness of systemic complications in patients with hidradenitis suppurativa.

A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration. In a press release on Wednesday, the FDA announced it had approved Casgevy, the first gene therapy for children with sickle cell disease. (NewsNation) — A new treatment for children aged 2 or older with sickle cell disease has been approved by the Food & Drug Administration (FDA). In a Wednesday news release, the FDA announced it had approved Casgevy, the first gene therapy for children with the disease. “Casgevy is a gene therapy consisting of the patient’s own (autologous) hematopoietic (blood) stem cells, administered as a one-time single dose for intravenous infusion,” the release noted. “Pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases,” Karim Mikhail, the acting director of the Center for Biologics Evaluation and Research, wrote. “These disorders carry a heavy burden for children and their families, affecting growth, development, and long-term health in profound ways,” Megha Kaushal, acting deputy director of the Office of Therapeutic Products in CBER, said in the release.