Newborn Screening, Enzyme Delivery, and the Future of Pompe Disease Treatment
May 14th 2025Priya Kishnani, MD, division chief of Medical Genetics at Duke University, reflects on Pompe disease’s historical and scientific evolution, from early enzyme therapy to next-generation treatment strategies and newborn screening breakthroughs.
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]
New Treatments, Global Collaboration Highlight 2025 Peripheral Nerve Society Annual Meeting
May 13th 2025Committee chairs Vincent Timmerman, PhD, MSc, and Kathrin Doppler, MD, provide a preview of the 2025 PNS Annual Meeting, giving clinicians an inside look at what to expect from the upcoming conference.
Illuminating Genetic Insights in Parkinson Disease Through PD GENEration: James Beck, PhD
April 30th 2025The chief scientific officer of the Parkinson’s Foundation talked about how broad genetic testing through PD GENEration is uncovering valuable prognostic and diagnostic insights in Parkinson disease. [WATCH TIME: 4 minutes]
Advancing NMOSD Care for Patients: Treatment Access, Education, and Holistic Well-Being
April 28th 2025Sumaira Ahmed, founder and executive director of The Sumaira Foundation, discussed how FDA-approved therapies transformed the NMOSD treatment landscape and highlighted ongoing challenges in patient care.
Prioritizing Brain Health in NMOSD Beyond Disease-Modifying Therapies: Sumaira Ahmed
April 27th 2025The founder and executive director of the Sumaira Foundation emphasized the critical role of lifestyle factors such as sleep, exercise, and nutrition for maintaining brain health in patients with NMOSD. [WATCH TIME: 3 minutes]
Durability and Next Steps in Advancing ARCUS Gene Editing Platform in DMD: Cassandra Gorsuch, PhD
April 26th 2025The chief scientific officer at Precision Biosciences provided clinical insights on the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Unveiling ARCUS Gene Editing Platform and Its Potential in DMD: Cassandra Gorsuch, PhD
April 23rd 2025The chief scientific officer at Precision Biosciences provided clinical commentary on ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy. [WATCH TIME: 4 minutes]
Breaking Down Oculopharyngeal Muscular Dystrophy: From Clinical Gaps to Genetic Gains
April 22nd 2025Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, offered a clinical and translational overview of OPMD, highlighting current care challenges and the early promise of dual-action gene therapy strategies.
Rethinking Alzheimer Diagnosis and Disease Staging: Clifford Jack, MD
April 21st 2025The neuroradiologist at Mayo Clinic discussed the clinical and conceptual differences between Alzheimer disease diagnostic frameworks and how clinicians should prepare for a shifting treatment landscape. [WATCH TIME: 4 minutes]
Evaluating Safety and Early Outcomes of Lecanemab Treatment for Alzheimer Disease: Philip Kuball, MD
April 18th 2025The resident in the Department of Neurology at NYU Langone Health discussed the preliminary findings of a 9-month study on lecanemab recently presented at the 2025 AAN Annual Meeting. [WATCH TIME: 2 minutes]
Michael Flanagan, PhD, on Del-zota’s Breakthrough Potential for Duchenne Muscular Dystrophy
April 17th 2025The chief scientific officer at Avidity Biosciences details the mechanism, early results, and future plans for Del-zota, an investigational therapy targeting exon 44 in Duchenne muscular dystrophy.
Celebrating Innovation in MS Research With the John Dystel Prize: Bruce Bebo, PhD
April 17th 2025The executive vice president of research at the National MS Society talked about how the award continues to honor transformative multiple sclerosis research and inspire the next generation of investigators. [WATCH TIME: 5 minutes]
Expanding Access to Whole Genome Sequencing in Parkinson Research: James Beck, PhD
April 17th 2025The chief scientific officer of the Parkinson’s Foundation discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations. [WATCH TIME: 4 minutes]