Video Series

Video 6 - "Current Front-Line Targeted Treatments in Ph+ ALL"

Elias Jabbour, MD, provides an overview of the history of targeted agents and the use of tyrosine kinase inhibitors (TKIs) in the treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

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.

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.