Outlining Phase 3 VITALIZE and MOBILIZE Trials of Riliprubart in CIDP: Luis Querol, MD, PhD
May 21st 2025The neurologist at the Hospital de la Santa Creu, in Barcelona, Spain, outlined the goals and significance of the VITALIZE and MOBILIZE phase 3 trials evaluating riliprubart in standard-of-care and treatment-refractory CIDP populations. [WATCH TIME: 3 minutes]
Place of Continuous Subcutaneous Infusion Systems in Parkinson Disease Management
Panelists discuss how continuous subcutaneous infusion systems should be strategically positioned within Parkinson disease management, typically as options for patients with advanced disease experiencing motor fluctuations despite optimized oral therapy.
Newborn Screening and Primary Care Awareness for Timely Referral
May 21st 2025An expert discusses strategies to lower the age of Duchenne muscular dystrophy (DMD) diagnosis, emphasizing the importance of universal newborn screening with the testing of creatine kinase (CK) levels, clinician recognition of motor red flags such as the Gower sign, early neurology referrals, and incorporating CK level testing in cognitive delay evaluations to enable timely therapeutic intervention and improve long-term outcomes.
Early Recognition of Duchenne Muscular Dystrophy: Key Signs Across Developmental Domains
May 21st 2025An expert discusses the typical presentation and diagnosis of Duchenne muscular dystrophy (DMD) around ages 5 to 6, highlighting early signs such as motor delays, Gower sign, and developmental concerns, while emphasizing the importance of newborn screening, clinician awareness, and developmental monitoring to enable earlier intervention and improved outcomes.
Hormonal Influences on Sleep Disorders in Women: Suzanne Bertisch, MD
May 21st 2025The chair of the Women’s Sleep Health Task Force at the American Academy of Sleep Medicine talked about how hormonal changes across a woman’s lifespan influence the prevalence of sleep disorders. [WATCH TIME: 4 minutes]
Continuous Subcutaneous Infusion Systems for Parkinson Disease and Patient Selection Criteria
Panelists discuss how the newly FDA-approved continuous subcutaneous levodopa infusion system (foscarbidopa/foslevodopa) offers another advanced treatment option, exploring the ideal candidates for these continuous infusion therapies based on disease characteristics, previous treatment responses, and patient preferences.
Biomarker and Subgroup Insights on Novel CIDP Therapy Riliprubart: Luis Querol, MD, PhD
May 20th 2025The neurologist at the Hospital de la Santa Creu, in Barcelona, Spain, provided clinical context on sustained responses and new biomarker findings from exploratory phase 2 data on riliprubart in CIDP. [WATCH TIME: 4 minutes]
A More Selective Immune Approach to CIDP With Riliprubart: Claudia Sommer, MD
May 19th 2025The president-elect of the Peripheral Nerve Society provided clinical context the mechanism and clinical relevance of riliprubart, a targeted complement inhibitor in development for chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 3 minutes]
Emerging Agents in Alzheimer’s Disease
May 19th 2025Panelists discuss how emerging therapeutic targets, including amyloid-beta plaques, tau protein aggregation, inflammation modulation, and mitochondrial dysfunction, offer promising approaches for disease-modifying treatments in early Alzheimer's disease.
Biomarkers in Alzheimer’s Disease (May 2025)
May 19th 2025Panelists discuss how recent advances in Alzheimer’s disease, including amyloid-targeting therapies like lecanemab and donanemab, along with the growing role of blood-based biomarkers for early diagnosis and monitoring, are transforming treatment paradigms, enabling disease modification and personalized care while improving accessibility and reducing barriers to diagnosis.
Donanemab for Treatment of Alzheimer’s Disease
May 19th 2025Panelists discuss how the TRAILBLAZER-ALZ-2 trial demonstrates that donanemab significantly reduces amyloid plaques and slows cognitive and functional decline in early Alzheimer’s disease, with a manageable safety profile and the potential for faster plaque removal compared to other amyloid-targeting therapies.
Building Consistency in Long-Term Management of Insomnia
May 16th 2025A panelist discusses how long-term success in treating chronic insomnia depends on patient adherence, realistic expectations, and a strong therapeutic relationship that supports consistent behavioral changes and ongoing treatment adjustments.
Managing Insomnia: History, Comorbidities, and Clinical Insight
May 16th 2025A panelist discusses how effective management of chronic insomnia requires a multifactorial, individualized approach that combines thorough patient evaluation with both behavioral strategies and targeted pharmacologic treatments.
Defining Rapid and Early Seizure Termination (REST) in Epilepsy
May 16th 2025A panelist discusses how REST empowers patients and caregivers to intervene at seizure onset with fast-acting rescue therapies, preventing escalation, reducing emergency care, and improving long-term outcomes and quality of life.
Recognizing Scientific Excellence and Impact in Multiple Sclerosis Research: Bruce Bebo, PhD
May 15th 2025The executive vice president of research at the National MS Society talked about the significance of the Dystel Prize, honoring researchers in MS whose scientific work has led to meaningful diagnostic or therapeutic advancements. [WATCH TIME: 2 minutes]
Advancing MS Care Through Early Intervention and Deeper CNS Insights: Jack P. Antel, MD
May 15th 2025The professor of neurology and neurosurgery at McGill University discussed the evolution of multiple sclerosis treatment, the need for earlier intervention, and the pressing scientific questions surrounding neuroinflammation. [WATCH TIME: 4 minutes]
Second-Line Treatments for CIDP
May 15th 2025Panelists discuss how treatment options for patients with chronic inflammatory demyelinating polyneuropathy (CIDP) who do not respond to intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) include second-line therapies such as plasmapheresis, corticosteroids, immunosuppressive agents, and newer options like efgartigimod, with a focus on a tailored approach to manage refractory cases effectively and improve patient outcomes.
Evaluating Efficacy and Individualizing Treatment in CIDP
May 15th 2025Panelists discuss how evaluating the efficacy of treatment in chronic inflammatory demyelinating polyneuropathy (CIDP) involves a combination of clinical, functional, and electrophysiological measures, and emphasize the importance of individualizing treatment approaches based on disease subtype, prior therapy response, comorbidities, patient preferences, and ongoing monitoring to optimize patient outcomes.
Advancing ALS Through Gene Therapies, Early Intervention, and Multidisciplinary Care: Brian Lin, PhD
May 15th 2025The research portfolio director at the Muscular Dystrophy Association discussed the evolving ALS therapeutic landscape, highlighting advances in genetic and RNA-based interventions. [WATCH TIME: 6 minutes]
The Critical Role of Dystrophin in the Pathophysiology of Duchenne Muscular Dystrophy
May 14th 2025An expert discusses the critical role of dystrophin in maintaining muscle integrity across multiple systems, explaining how its absence in Duchenne and Becker muscular dystrophy leads to widespread complications—including neurologic, pulmonary, cardiac, gastrointestinal, urinary, and orthopedic issues—driven by chronic muscle damage, inflammation, and fibrosis.
Navigating Duchenne Muscular Dystrophy: An Early-Childhood Case Example
May 14th 2025An expert discusses the evolving treatment landscape of Duchenne muscular dystrophy (DMD), highlighting advances in gene therapy, exon-skipping agents, and novel corticosteroids that aim to slow disease progression, improve muscle function, and enhance quality of life, while emphasizing the growing role of personalized medicine and early intervention in optimizing long-term outcomes.
CSAI and INFUS-ON Study for Treatment of Parkinson Disease
Panelists discuss how the recently FDA-approved SPN-830 apomorphine infusion pump demonstrated significant efficacy in reducing off time and improving motor function in the INFUS-ON study, with physicians expressing optimistic views about both the US and European clinical trial results.
Continuous Subcutaneous Apomorphine Infusion (CSAI) and TOLEDO Study in Parkinson Disease
Panelists discuss how continuous subcutaneous apomorphine infusion (CSAI) demonstrates favorable pharmacokinetics compared with other formulations, with the TOLEDO study and its extension showing significant reductions in off time and improvements in motor function with manageable safety profiles.
Insights on Quanterix Simoa Assay to Diagnose Alzheimer Disease: Mark Roskey, PhD
May 13th 2025The chief scientific officer at Quanterix gave a clinical overview on how the company’s Simoa platform enables ultrasensitive detection of neurodegeneration biomarkers in blood, transforming Alzheimer and Parkinson disease research. [WATCH TIME: 3 minutes]
A Mother’s Journey Through Diagnosis and Acceptance of Duchenne Muscular Dystrophy: Debra Miller
May 13th 2025The chief executive officer and founder at CureDuchenne shared her emotional journey from her son’s diagnosis of Duchenne to embracing a new path focused on advocacy, adaptation, and resilience. [WATCH TIME: 4 minutes]