Patient Advocates Demand Oral Parity in North Carolina

A recent study suggests the differences between ambulation definitions for patients with Duchenne muscular dystrophy (DMD) can impact the identification of ambulant vs nonambulant individuals, and standard criteria across settings are needed.

Jun Gong, MD, of Cedars-Sinai Medical Center, discusses the latest data on adagrasib in colorectal cancer, the importance of conducting genomic testing, and more.

Government agencies have created an online portal for the public to report potential anticompetitive practices in health care; there are changes coming to the “boxed warning” section for chimeric antigen receptor T-cell therapies (CAR T) to highlight T-cell blood cancer risk; questions about the safety of obesity medications during pregnancy have arisen in women on them who previously struggled with fertility issues.

Justin Bekelman, MD, director of the Penn Center for Cancer Care Innovation, sat with our hosts Emeline Aviki, MD, MBA, and Stephen Schleicher, MD, MBA, for our final episode of 2023 to discuss the importance of collaboration between academic medicine and community oncology and testing innovative cancer care delivery in these settings.

In their preclinical model, the researchers found efficacy both in vitro and in vivo by using CRISPR-Cas9 to mimic porphyria and combining the technology with light therapy.

A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).