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Evaluating Transformative Gene Therapies

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. ....

Kimberlyc Chen , Mary Pak , Ryan Haumschild , Emma Ciafaloni , Peer Exchange , Jessica Nance , Gene Therapy , Managed Care , Patient Access , Clinical Data , Rare Diseases , Treatment Options , Ethical Implications , Cost Effectiveness , Coverage Determinations , Clinical Evidence , Patient Population , Financial Sustainability , Subscription Models , Warranty Programs , Performance Based Contracts , Value Based Contracting , Patient Advocacy , Research And Development , Innovative Therapies , Data Sharing ,

Viral Vectors in Gene Therapy

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

Mary Pak , Ryan Haumschild , Emma Ciafaloni , Kimberlyc Chen , Peer Exchange , Jessica Nance , Gene Therapy , Managed Care , Patient Access , Clinical Data , Rare Diseases , Treatment Options , Ethical Implications , Cost Effectiveness , Coverage Determinations , Clinical Evidence , Patient Population , Financial Sustainability , Subscription Models , Warranty Programs , Performance Based Contracts , Value Based Contracting , Patient Advocacy , Research And Development , Innovative Therapies , Data Sharing ,

Overview of Gene Therapy Approaches and Impacts

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. ....

Emma Ciafaloni , Mary Pak , Kimberlyc Chen , Ryan Haumschild , Peer Exchange , Jessica Nance , Gene Therapy , Managed Care , Patient Access , Clinical Data , Rare Diseases , Treatment Options , Ethical Implications , Cost Effectiveness , Coverage Determinations , Clinical Evidence , Patient Population , Financial Sustainability , Subscription Models , Warranty Programs , Performance Based Contracts , Value Based Contracting , Patient Advocacy , Research And Development , Innovative Therapies , Data Sharing ,

Key Factors in Gene Vector Selection

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. ....

Mary Pak , Kimberlyc Chen , Emma Ciafaloni , Ryan Haumschild , Peer Exchange , Jessica Nance , Gene Therapy , Managed Care , Patient Access , Clinical Data , Rare Diseases , Treatment Options , Ethical Implications , Cost Effectiveness , Coverage Determinations , Clinical Evidence , Patient Population , Financial Sustainability , Subscription Models , Warranty Programs , Performance Based Contracts , Value Based Contracting , Patient Advocacy , Research And Development , Innovative Therapies , Data Sharing ,

Lawmaker proposes restricting use of AI in Georgia healthcare, insurance coverage decisions

A member of the Georgia House of Representatives filed legislation to ban the use of artificial intelligence programs when making insurance, health coverage, or public assistance award decisions. ....

United States , Mandisha Thoma , Georgia House , House Bill , Georgia Composite Medical Board , Health Coverage , Coverage Determination , Coverage Determinations , Georgia Composite Medical Board , Artificial Intelligence , Public Assistance ,