Video Series

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North Star Ambulatory Assessment, time to rise from floor, and 10-m walk/run compared with external control groups over 2 years of treatment.

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer technologies, while highlighting the ongoing challenges of identifying at-risk populations and collecting comprehensive safety and efficacy data.

Panelists discuss how treating obesity as the root cause can create a "virtuous cycle" where weight loss improves insulin resistance, reduces medication needs, and enables better physical activity, breaking the traditional reactive approach to diabetes management.

Panelists emphasize that patients with KMT2A-rearranged acute myeloid leukemia (AML) often face steep educational challenges at diagnosis, requiring clear communication about the subtype’s adverse-risk classification, biological complexity, and treatment implications—highlighting the need for accessible education, academic-community collaboration, and early referral to specialized care for optimal outcomes.

A panelist discusses how dry eye disease involves tear film imbalance, leading to inflammation and ocular surface damage, emphasizing the importance of precise diagnosis and tailored treatment while acknowledging ongoing challenges with patient expectations, medication access, delayed symptom relief, and treatment adherence.

Panelists explain that KMT2A-rearranged acute myeloid leukemia (AML) is an aggressive, molecularly distinct leukemia subtype marked by menin-dependent transcriptional dysregulation, poor response to standard therapy, diagnostic challenges, and high relapse risk—underscoring the urgency of RNA-based diagnostics, early transplant consideration, and emerging menin-targeted treatments to improve outcomes.

Panelists discuss the critical role of psychosocial support in managing NF1-associated plexiform neurofibromas, emphasizing the need for psychotherapy, counseling, and social work interventions to address anxiety, depression, and social stigma while also highlighting challenges in accessing therapies like selumetinib due to cost, insurance barriers, and health care system disparities.

Panelists discuss how treatment strategies differ between transplant-eligible and transplant-ineligible multiple myeloma patients, with transplant eligibility determined by functional status rather than age alone, and how quadruple therapy regimens are increasingly used for transplant-eligible patients to achieve deep, durable responses.

A panelist discusses how bleeding risk assessment depends on platelet count levels (highest risk below 20,000), requiring careful medication history review to avoid drugs that impair platelet function like aspirin and NSAIDs.

Panelists discuss how autologous stem cell transplant remains essential in frontline multiple myeloma management despite improved quadruple therapy outcomes, emphasizing that current evidence still supports transplantation for eligible patients rather than deferring it.

Panelists discuss how barriers to implementing evidence-based obesity and diabetes care include limited provider time, lack of multidisciplinary teams, insufficient education about obesity physiology, and high costs of medications and programs.

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest efficacy with fewer systemic adverse effects than intravenous administration, though safety concerns and limited data remain.

Panelists discuss how current therapeutic strengths include 15 approved treatments with different mechanisms of action that can work synergistically, while limitations involve variable delivery methods and complexity, leading to more aggressive upfront combination therapy approaches.

Panelists discuss how policy makers can increase biosimilar adoption through financial incentives, streamlined interchangeability rules, and addressing patent litigation delays, emphasizing that biosimilar savings will ultimately help fund the next wave of health care innovation such as cell and gene therapies.

Panelists discuss how comorbidities such as scoliosis, dislocated hips, and nutritional issues affect spinal muscular atrophy (SMA) treatment decisions, with treatment approaches evolving as patients' functional abilities improve with disease-modifying therapies.

Panelists discuss how biosimilar manufacturers can increase uptake by ensuring positive financial outcomes for all stakeholders, maintaining strong patient assistance programs, and working closely with health care providers who significantly influence biosimilar implementation.

Panelists discuss how treatment individualization depends on patient-specific factors including age, comorbidities, disease severity, and personal preferences, balancing clinical guidelines with shared decision-making to optimize both efficacy and adherence.

Panelists discuss how subcutaneous (SubQ) drug formulations offer practical value by significantly reducing treatment times, easing staffing pressures, and enhancing efficiency in oncology practices, ultimately supporting a more scalable and sustainable care model.

Panelists discuss how the shift from intravenous (IV) to subcutaneous (SubQ) drug administration is transforming oncology care delivery, highlighting its lasting impact on patient experience, health care efficiency, and clinical workflows in light of recent FDA approvals and evolving pharmaceutical strategies.

Panelists discuss how bronchiectasis is more common than previously thought, with growing awareness, research, and specialized centers improving diagnosis and treatment options, though challenges remain in standardizing care and securing insurance coverage for therapies.

Panelists discuss how effective management of idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) relies on early, accurate diagnosis and multidisciplinary collaboration, with a focus on personalized treatment strategies, proactive adverse effect management, and supportive interventions like pulmonary rehabilitation and nutrition to enhance quality of life and improve long-term outcomes.

Panelists discuss how managing idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) requires a comprehensive, patient-centered approach that addresses not only physiological decline but also the significant psychological, social, and functional burdens patients face—highlighting the need for early diagnosis, emotional support, and coordinated care to improve both quality of life and health care outcomes.

Panelists discuss how future bronchiectasis research will focus on precision medicine, identify treatable traits, and expand networks of Centers of Excellence to provide advanced care and clinical trial access for patients.

Panelists discuss how the Inflation Reduction Act (IRA) might influence health care systems’ selection and utilization of Bruton tyrosine kinase (BTK) inhibitors, particularly through changes in pricing, reimbursement policies, and treatment access.

Panelists discuss how lower out-of-pocket costs resulting from ibrutinib’s inclusion in the Inflation Reduction Act (IRA) might improve the affordability and utilization of BTK inhibitor treatments in chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), potentially expanding access for patients.

Panelists discuss how the management of immunoglobulin A (IgA) nephropathy is undergoing a transformative shift from symptom control to targeted disease modification, with emerging therapies offering new hope for patients while requiring thoughtful integration into clinical practice and health care systems.

Panelists discuss how economic considerations in immunoglobulin A (IgA) nephropathy medication management vary dramatically across chronic kidney disease (CKD) stages, with early intervention costs being modest but potentially cost-saving compared with the extraordinary expenses of advanced disease management and renal replacement therapy.

Panelists discuss how promising ongoing and upcoming clinical trials may reshape sequencing strategies in ALK+ non–small cell lung cancer (NSCLC).

Panelists discuss how immunoglobulin A (IgA) nephropathy’s progressive nature and significant economic impact demand a shift in payer perspective toward investing in disease-modifying therapies that, despite higher upfront costs, can prevent the extraordinary expenses of dialysis and transplantation while preserving patients’ quality of life and productivity.

Panelists discuss how to approach patient education after progression on an ALK inhibitor, address real-world challenges during therapy transitions, and determine best options for patients progressing on lorlatinib.