Video Series

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein expression, including 2 requiring ongoing treatment and 1 gene transfer therapy (onasemnogene abeparvovec) approved only for patients under 2 years of age.

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and less deterioration compared with natural history, providing confidence in the therapy’s long-term benefits while acknowledging the need for survival data and addressing concerns about potential transgene dilution over time.

Panelists discuss how implementing cardiovascular-kidney-metabolic health requires a team-based approach using the analogy of football, where physicians coach multidisciplinary teams to help patients overcome barriers and achieve health goals.

Panelists highlight that menin inhibitors represent a breakthrough in the treatment of KMT2A-rearranged AML by targeting the disease’s core epigenetic drivers, with early clinical trials like AUGMENT-101 showing promising results in heavily pretreated, high-risk patients and supporting the potential for a new therapeutic standard in this aggressive leukemia subtype.

Panelists discuss how minimal residual disease (MRD) status can guide post-transplant treatment decisions, particularly for high-risk patients who don’t achieve MRD negativity and patients considering discontinuation of long-term maintenance therapy.

Panelists explain that KMT2A-rearranged acute myeloid leukemia (AML) is a biologically aggressive and diagnostically complex leukemia subtype—especially prevalent in pediatric and therapy-related cases—with variable prognostic implications depending on fusion partners, underscoring the need for early molecular identification, personalized treatment strategies, and expanded access to targeted therapies and advanced diagnostics.

A panelist discusses how ITP creates a chronic inflammatory state causing underrecognized symptoms like fatigue and joint pain that significantly impact patients' quality of life and ability to function, while recommending avoidance of activities that could cause head or internal injuries.

Panelists discuss key lessons learned from previous NF1-associated plexiform neurofibroma therapies, including the limitations of traditional treatments due to high toxicity and limited efficacy, and how these challenges are driving the development of more targeted therapies like MEK inhibitors, personalized medicine, and combination strategies to improve outcomes and reduce relapse.

Panelists discuss how GLP-1 receptor agonists represent a paradigm shift by providing weight loss, glycemic control, and cardiovascular protection simultaneously, moving focus beyond A1C control to comprehensive risk reduction.

Panelists discuss how quadruplet regimens show improved efficacy over triplet regimens without significantly increased safety concerns, with anti-CD38 antibodies being well tolerated and NCCN guidelines now recommending 4-drug regimens for transplant-eligible patients.

A panelist discusses how the COAT trial demonstrated that twice-daily 0.09% cyclosporine improves multiple signs and symptoms of dry eye disease within 28 days, highlighting the value of early monitoring to optimize patient adherence and treatment outcomes.

Panelists discuss how insurance challenges impact spinal muscular atrophy (SMA) care, with prior authorizations becoming more streamlined over time but limitations on physical therapy sessions and inequitable access to treatments remain significant barriers.

Panelists discuss how sotatercept represents a paradigm shift as the first activin signaling inhibitor showing dramatic improvements in heavily pretreated patients, with the Stellar and Zenith trials demonstrating significant reductions in clinical worsening and potential for reverse remodeling.

Panelists discuss how riociguat targets the nitric oxide pathway and is approved for both Group 1 PAH and Group 4 chronic thromboembolic pulmonary hypertension, with specific patient selection criteria based on risk stratification and surgical candidacy.

Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments, hold promise for addressing fatigue and improving muscle function in older patients.

Panelists discuss how subcutaneous (SubQ) oncology treatments are reshaping clinic operations by improving infusion chair utilization, enabling flexible scheduling, streamlining pharmacy workflows, and prompting new care delivery models that enhance both efficiency and patient experience.

Panelists discuss several promising new agents for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF), including an LPA1 receptor antagonist showing lung function preservation, integrin inhibitors targeting fibrotic pathways despite safety challenges, and inhaled prostaglandins being evaluated as a well-tolerated add-on therapy in ongoing trials.

Panelists discuss how antifibrotic therapies for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) slow disease progression despite challenges with cost, tolerability, and limited symptom relief, emphasizing the importance of patient education and aligning treatment goals.

A panelist discusses the latest advancements presented at ASCO 2025, highlighting talquetamab’s pivotal trial in relapsed/refractory multiple myeloma that demonstrated high response rates and longer durability with biweekly dosing, including in patients previously treated with B-cell maturation antigen–targeted therapies, supporting sequencing and combination strategies to improve outcomes.

Panelists discuss the specific challenges and barriers clinicians might encounter when utilizing different BTK inhibitors under the regulations of the Inflation Reduction Act (IRA), including issues related to cost, access, and treatment guidelines.

Panelists discuss potential changes to clinical pathways for Bruton tyrosine kinase (BTK) inhibitor therapies as a result of the Inflation Reduction Act (IRA), focusing on how shifting costs and access regulations may impact treatment decisions and patient care strategies.

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.