Video Series

Video 4 - "Screening and Diagnosing of Ph+ ALL in Adults"

The panel discusses optimal strategies for screening and detecting Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in patients across different treatment stages.

Aaron Gerds, MD, MS, explores the future landscape of myelofibrosis with insights from the 2003 ASH Annual Meeting and Exposition (ASH 2023), and delves into anticipated data on combination therapies and ongoing trials shaping the next phase of myelofibrosis treatment.

Jessica Nance, MD, MS, reviews the data and evidence that led to accelerated approval of delandistrogene moxeparvovec-rokl for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the transformative potential of gene therapies.

Vivian Tambe Ebot-Tar, PharmD, MBA, guides a discussion on the incidence of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) and how payers can have the most impact through early treatment coverage.

Gain insights into valuable educational resources for patients coping with desmoid tumors, such as global consensus papers and advocacy group websites, and the significance of seeking multiple expert opinions for informed decision-making.

Delve into the impact of observing desmoid tumors and strategies for maintaining patient well-being during treatment, and learn about the importance of education, shared decision-making, and balancing conservative vs aggressive treatment.

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.

Explore effective approaches in handling fatigue, insomnia, and financial difficulties of patients with desmoid tumors as experts highlight psychosocial support and treatment considerations for quality care.

Aaron Gerds, MD, MS, explores the FDA approval of momelotinib and its swift integration into National Comprehensive Cancer Network (NCCN) guidelines. He reviews different JAK inhibitors based on adverse effects and treatment goals, addressing potential treatment-related anemia in myelofibrosis.

Explore the multifaceted impact of desmoid tumors on patients’ physical, functional, and psychosocial well-being as experts emphasize effective symptom management and quality-of-life enhancements.

Kimberly C. Chen, DO, MSHLM, dives into the role of genetic testing in identifying patients eligible for gene therapy and explores coverage criteria, clinical validity, and utility considerations crucial for payers and managed care executives.

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

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