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A panel of experts discuss improving collaboration between health systems and managed care organizations to ensure optimal outcomes for patients with rare diseases.

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.

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.

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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.

Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease.

Ryan Haumschild, PharmD, MS, MBA, and Mary Pak, MD, FACP, consider FDA-approved gene therapies for hemophilia, spinal muscular atrophy, and Duchenne muscular dystrophy, exploring their genetic foundations, the FDA approval process, and considerations for therapeutic value and cost.

Emma Ciafaloni, MD, FAAN, describes the key differences among viral vectors used in gene therapy, highlighting their applications and strengths in targeting specific diseases.

Experts delve into the fundamentals of gene therapy, covering in vivo and ex vivo methods, and explore cellular function targets for effective treatment of a disease.

Jessica Nance, MD, MS, discusses maximizing gene therapy efficiency by selecting vectors for gene size, tissue targeting, and low exposure, and focusing on precise promoters for expression.