Recognizing AD Across All Skin Tones
August 28th 2025Panelists discuss how atopic dermatitis (AD) presents differently across skin tones, appearing as purple, gray, or barely visible inflammation rather than classic redness, with perifollicular prominence and postinflammatory pigmentation changes being more prominent in patients with darker skin.
Beyond the Itch: The Full Spectrum of AD Symptoms and Their Impact on Patients
August 28th 2025Panelists discuss how atopic dermatitis (AD) extends far beyond pruritus to include pain, sleep disturbances, psychosocial stigma, and quality-of-life impacts that affect patients’ work, school, and daily functioning regardless of disease severity.
Bridging the Gaps: Health Care Professional Knowledge Gaps in AD Pathophysiology
August 21st 2025Panelists discuss how health care professionals face educational gaps in understanding atopic dermatitis (AD) immunopathogenesis, with advanced practice providers (APPS) particularly needing additional training outside standard curricula to master biologic therapy selection.
The Cost of Waiting: Unlocking Treatment in a Timely Manner for MS Patients
August 15th 2025Panelists discuss how providers and payers must collaborate to develop innovative coverage criteria for BTK inhibitors that prevent patients with subclinical progression from being forced to fail multiple inappropriate therapies before accessing optimal treatment.
Modeling the Cost of Progression: Capturing the True Burden of MS
August 8th 2025Panelists discuss how economic models must account for the broader impact of MS progression on earning potential, family planning, caregiver burden, and quality of life rather than focusing solely on direct medical costs.
The Value of Functional Outcomes and Elevating Patient Voices in MS Assessments
August 1st 2025Panelists discuss how patient-reported outcomes are crucial for capturing MS symptoms like fatigue, depression, and cognitive decline that significantly impact working-age patients but require standardization for practical clinical integration.
Evaluating the Future of MS Therapies: Balancing Early Data With Delayed Outcomes
July 25th 2025Panelists discuss how the evaluation process must evolve to accommodate therapies that show benefits in slowing atrophy and motor decline over 1 to 2 years, emphasizing the need for early coverage rather than waiting for extensive long-term data.
Final Words: Aligning Innovation, Guidelines, and Access
July 18th 2025Panelists expressed cautious optimism about high-risk acute myeloid leukemia (AML) treatment, emphasizing the need for ongoing research, personalized therapy based on molecular profiling, and strengthened collaboration between community and academic centers to improve patient outcomes, while recognizing that education and sharing best practices are key to advancing targeted therapies and achieving long-term cures.
The Power of Advocacy: Supporting Patients with KMT2A AML
July 18th 2025Panelists highlight that patient advocacy is crucial in improving high-risk acute myeloid leukemia (AML) outcomes by providing emotional support, funding research, influencing clinical guidelines, promoting patient engagement, facilitating collaboration between community and academic care, addressing systemic access barriers, and implementing innovative programs like medication recycling to enhance treatment availability.
Bridging the Gaps: BTK Inhibitors and the Future of MS Care
July 18th 2025Panelists discuss how BTK inhibitors represent a promising new oral therapy class that could address both inflammatory and neurodegenerative aspects of MS, particularly for progressive forms where treatment options are limited.
Accessing Innovation: Practical Tips and Policy Hurdles
July 18th 2025Panelists advocate for a streamlined approach to diagnosing KMT2A-rearranged acute myeloid leukemia (AML) that includes standardized testing protocols (cytogenetics, fluorescence in situ hybridization [FISH], and next-generation sequencing) at diagnosis, close collaboration with laboratories to clarify complex reports, adoption of rapid testing methods, and flexible sample collection strategies to ensure timely, accurate identification and optimized patient care.
MD Treatment From a Managed Care Perspective
July 14th 2025Panelists discuss how payers seek good return on investment when evaluating expensive gene therapies, creating potential friction when innovative treatments come at significant costs, requiring ongoing dialogue between manufacturers, patients, payers, and physicians to determine appropriate value and access.
Delays in Detection and the Impact on Access to Targeted Therapy
July 11th 2025Panelists highlight that despite established guidelines, delays and gaps in molecular profiling—especially for KMT2A rearrangements—persist due to report complexity, misinterpretation, and misconceptions about patient eligibility, underscoring the urgent need for improved provider education, expert collaboration, and comprehensive testing to ensure accurate diagnosis, optimal treatment selection, and better patient outcomes in acute myeloid leukemia (AML).
Aligning Evidence With Access: Disability Outcomes, Data, and Payer Decision-Making
July 11th 2025Panelists discuss how emerging biomarkers like neurofilament light protein, glial fibrillary acidic protein (GFAP), cervical cord atrophy, and phase rim lesions could enable earlier identification of patients at risk for progression, supporting more effective treatment decisions and payer coverage determinations based on longitudinal disability data rather than just relapse activity.
The Why and When of Molecular Testing in AML
July 11th 2025Panelists stress that timely and accurate diagnosis of KMT2A-rearranged acute myeloid leukemia (AML) is essential for personalized treatment planning, highlighting the need to overcome systemic delays in molecular testing, foster academic-community collaboration, and educate both clinicians and patients on the safety and importance of waiting for complete genetic data before initiating therapy.
Looking to the Future in MD Treatment
July 7th 2025Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down rather than restore gene expression for these autosomal dominant conditions.
Closing the Gaps: Therapeutic Performance and the Promise of Innovation
July 4th 2025Panelists discuss how current multiple sclerosis (MS) therapies show limited effectiveness against progression, but emerging Bruton tyrosine kinase (BTK) inhibitors offer promise by targeting both B cells and central nervous system (CNS)–penetrating microglia, with one showing a 31% reduction in confirmed disability progression in clinical trials.
Emerging Menin Inhibitors and Patient Impact
July 4th 2025Panelists highlight that the emergence of menin inhibitors is reshaping the treatment paradigm for KMT2A-rearranged acute myeloid leukemia (AML), with ongoing research focused on optimizing their use, overcoming resistance, expanding access, and integrating these targeted therapies into personalized and potentially curative treatment strategies across diverse patient populations.
Digging Deeper: Mechanisms of Progression and the Case for a Dual Therapeutic Approach
July 4th 2025Panelists discuss how chronic neuroinflammation involves distinct mechanisms from acute relapses—including microglial activation, mitochondrial dysfunction, and iron deposition—necessitating dual therapeutic approaches that address relapsing and progressive disease components.
The Impact of Intrathecal Onasemnogene Abeparvovec Therapy in SMA
June 30th 2025Panelists discuss how intrathecal delivery of onasemnogene abeparvovec in the STEER study demonstrates statistically significant motor function improvements in older patients with SMA (ages 2-18 years) with favorable safety profiles, potentially expanding gene therapy access beyond the current age restriction of under 2 years.
Gaps in Care for Childhood SMA
June 30th 2025Panelists discuss how gaps in SMA care persist despite highly effective treatments, particularly regarding racial and ethnic disparities in research participation and global access challenges due to high costs and infrastructure limitations in developing countries.
Measuring What Matters: Evidence and End Points That Define Disability in MS
June 27th 2025Panelists discuss how robust clinical evidence from major studies like the EPIC and OPERA trials demonstrates that progression independent of relapse activity (PIRA) is the primary driver of confirmed disability progression in patients with multiple sclerosis (MS), with clinical parameters over 3 to 6 months being the most meaningful measures of treatment impact.
Shifts in Standard of Care for Adults and Children With KMT2A AML
June 27th 2025Panelists emphasize that the approval of menin inhibitors has transformed the treatment landscape for KMT2A-rearranged acute myeloid leukemia (AML) by replacing historically limited and toxic chemotherapy-based strategies with a targeted, guideline-endorsed option that aligns with disease biology—offering renewed hope for both adult and pediatric patients, especially in the relapsed/refractory setting.