Jessica Nance, MD, MS, and Emma Ciafaloni, MD, FAAN, discuss ethical dilemmas, exploring how patient subgroup restrictions on orphan drugs can impact access and cost control.
Health care professionals discuss the challenges related to developing and interpreting clinical data for rare disease therapies to guide payer coverage decisions.
Ryan Haumschild, PharmD, MS, MBA, leads a discussion on factors that influence coverage of non-oncology gene therapies, exploring the balance between long-term cost savings and high initial prices, patient selection, and the durability of effect for these therapies.
Forty health professionals, clinicians and journalists from France and Belgium attended a March 8 presentation and tour of Winship Cancer Institute at Emory Midtown.
Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early intervention and comprehensive care.