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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 chenMary pakRyan haumschildEmma ciafaloniPeer exchangeJessica nanceGene therapyManaged carePatient accessClinical dataRare diseasesTreatment optionsEthical implicationsCost effectivenessCoverage determinationsClinical evidence

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 pakRyan haumschildEmma ciafaloniKimberlyc chenPeer exchangeJessica nanceGene therapyManaged carePatient accessClinical dataRare diseasesTreatment optionsEthical implicationsCost effectivenessCoverage determinationsClinical evidence

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 ciafaloniMary pakKimberlyc chenRyan haumschildPeer exchangeJessica nanceGene therapyManaged carePatient accessClinical dataRare diseasesTreatment optionsEthical implicationsCost effectivenessCoverage determinationsClinical evidence

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 pakKimberlyc chenEmma ciafaloniRyan haumschildPeer exchangeJessica nanceGene therapyManaged carePatient accessClinical dataRare diseasesTreatment optionsEthical implicationsCost effectivenessCoverage determinationsClinical evidence

MSRvantage Introduces Blockchain Technology For A More Sustainable Future

MSRvantage Introduces Blockchain Technology For A More Sustainable Future
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Balaji kandregulaMsrvantage blockchainVice presidentPerformance based contracts