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Duchenne Muscular Dystrophy Treatment Landscape Review

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 , Kimberlyc Chen , Ryan Haumschild , Mary Pak , 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 ,

Shifting the Treatment Paradigm in Spinal Muscular Atrophy

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. ....

Kimberlyc Chen , Mary Pak , 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 ,

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 ,