SRNA Soundwaves

ABCs of NMOSD 701. University of Rochester NMO-Health Index Study

Krissy Dilger of SRNA spoke with Matt Rathbun and Charlotte Engebrecht from the University of Rochester Center for Health and Technology about the Neuromyelitis Optica Spectrum Disorder-Health Index (NMOSD-HI) study, which aims to develop and validate an NMOSD-specific patient-reported outcome survey for use in clinical trials and routine care [02:25]. They explained that existing measures are often adapted from multiple sclerosis and may not reflect NMOSD patients’ unique experiences [04:38]. They shared phase one insights from interviews with 15 individuals showing multi-system impacts [09:51]. They described eligibility for the current anonymous survey (adults 18+ with NMOSD, aquaporin-4 positive or negative, in the US, Canada, EU, UK, or Australia) and noted prior participants can join later phases [13:22]. You can learn more about the study here: https://redcap.link/nmo-hi Questions can be sent to Matt and Charlotte: Matthew_Rathbun@urmc.rochester.edu Charlotte_Engebrecht@urmc.rochester.edu Matt Rathbun, BA, graduated from Nazareth University in May of 2025 and is currently pursuing his Master of Public Health degree at Nazareth University. At the University of Rochester Center for Health + Technology (CHeT), he works as a Human Subject Research Specialist, where he coordinates translational research studies focused on the lived experiences of individuals living with rare diseases. This work supports the development and validation of disease-specific PRO measures that capture aspects of disease burden most meaningful to patients. Matt’s interests center on strengthening the relevance, inclusivity, and equity of clinical research. He aims to ensure that clinical research more accurately reflects the real-world impact of disease on patients’ lives. He also works to advance more equitable and patient-centered approaches to treatment evaluation in rare disease communities. Charlotte Engebrecht, BS, is a graduate of Hobart and William Smith Colleges and a current Master of Science in Clinical Investigations student at the University of Rochester. She serves as a Clinical Trials Project Specialist at the University of Rochester Center for Health + Technology (CHeT), where her work centers on the development and validation of patient-reported outcome (PRO) measures for rare diseases. Charlotte conducts research that is grounded in a commitment to elevating the patient voice as a central pillar of clinical research. Patient-reported outcomes offer critical insight into how diseases and treatments truly impact daily life. She is particularly passionate about ensuring that these perspectives are not only included, but prioritized, in the design and evaluation of clinical trials. Her work focuses on rare diseases, with a specific interest in neuromyelitis optica spectrum disorder (NMOSD), where traditional clinical endpoints often fail to capture the full burden of illness. By integrating patient-centered measurement tools into therapeutic development, Charlotte aims to advance more meaningful and responsive approaches to evaluating new treatments. 00:00 Welcome 02:25 Study Overview 04:38 Why Patient Voices Matter 06:16 How the Study Works 08:29 Who Can Participate 09:51 Phase One Findings 13:22 Join the Survey 15:43 Wrap Up

Community Meets Clinic 302. Drs. Grace Gombolay and Varun Kannan

The "Community Meets Clinic" podcast series introduces clinicians and healthcare personnel specializing in rare neuroimmune disorders. In this episode hosted by Krissy Dilger of SRNA, we met Dr. Grace Gombolay and Dr. Varun Kannan, both from Emory University and Children's Healthcare of Atlanta, designated Centers of Excellence in Rare Neuroimmune Disorders. Dr. Kannan discussed learning alongside families as conditions like MOG antibody disease emerged clinically and his focus on tailoring treatment and supporting clinical trials in a field with few approved therapies [03:37]. Dr. Gombolay outlined her research on biomarker development, a Children’s biobank, advanced MRI collaborations, and participation in the Network of Pediatric MS Centers covering disorders such as MOGAD, NMOSD, optic neuritis, ADEM, and TM [06:36]. They described their multidisciplinary clinic team, highlighted home infusions and telemedicine to reduce burden, and shared personal self-care strategies [10:22]. Dr. Gombolay and Dr. Kannan expressed hope for more trials, remyelination, prevention, and earlier diagnosis aided by AI prompts [20:43]. You can view Dr. Grace Gombolay's medical profile here: https://www.choa.org/doctors/grace-gombolay You can view Dr. Varun Kannan's medical profile here: https://www.choa.org/doctors/varun-kannan Grace Gombolay, MD, MSc, FAAN is an Associate Professor at Emory University and Director of the Pediatric Neuroimmunology and Multiple Sclerosis Clinic at Children's Healthcare of Atlanta. Her research interest involves biomarker development in pediatric neuroinflammatory diseases including autoimmune encephalitis, multiple sclerosis, MOGAD, and NMOSD. Varun Kannan, MD graduated from Emory University School of Medicine in 2017. He then completed child neurology residency in 2022, followed by pediatric neuroimmunology and multiple sclerosis fellowship at Baylor College of Medicine and Texas Children's Hospital in 2023. He returned to Emory and Children's Healthcare of Atlanta in 2023, where he has worked closely with Dr. Grace Gombolay in the neuroimmunology program. He is interested in clinical research regarding severe/relapsing forms of rare neuroimmune disorders including autoimmune encephalitis and MOGAD. He is currently involved in multiple upcoming phase 3 clinical trials exploring new disease modifying treatments for pediatric rare neuroimmune disorders. He is also passionate about medical education and is currently one of the Associate Program Directors for the Emory child neurology residency. 00:00 Welcome 01:56 Dr. Grace Gombolay's Journey 03:37 Dr. Varun Kannan's Path 05:06 Kannan's Research Focus 06:36 Biomarkers and Biobank 10:22 Clinic Team and Care 13:44 Self Care and Balance 16:15 Children's Healthcare of Atlanta 20:43 Hopeful Future Ahead 24:49 Closing

Ask the Expert 1404. MOGcast | The State of MOGAD Science

In this special “Ask the Expert” collaboration between The MOG Project and SRNA, Julia Lefelar and Dr. GG deFiebre welcomed Dr. Benjamin Greenberg of UT Southwestern, who answered questions from the audience. Dr. Greenberg reviewed major advances in MOG antibody disease research and diagnostic criteria [00:05:06]. He discussed efforts to predict relapse risk using sustained antibody positivity, demographic and clinical models, and immune-cell profiling studies [00:07:55]. Dr. Greenberg detailed controversies around low-positive antibody titers and how cell-based assays and dilution thresholds affect specificity [00:21:38]. He outlined concepts and progress in tolerance-inducing approaches such as Tregs and CAR T therapy, described differences from B-cell–depleting drugs like rituximab [00:26:32] Finally, Dr. Greenberg highlighted the satralizumab meteoroid trial and the ongoing cosMOG study of rozanolixizumab, emphasizing community engagement, registries, surveys, and trial participation to accelerate access and potential curative strategies [00:38:36]. You can learn more about The MOG Project here: https://mogproject.org/ Benjamin M. Greenberg, MD, MHS is a Professor and the Cain Denius Scholar in Mobility Disorders in the Department of Neurology [ https://utswmed.org/why-utsw/departments/neurology/ ] at UT Southwestern Medical Center in Dallas, Texas. He currently serves as the Vice Chair of Translational Research and Strategic Initiatives for the Department of Neurology. He is also the interim Director of the Multiple Sclerosis Center [ https://utswmed.org/locations/aston/multiple-sclerosis-and-neuroimmunology-clinic/ ] and the Director of the Neurosciences Clinical Research Center. In addition, he serves as Director of the Transverse Myelitis and Neuromyelitis Optica Program and the Pediatric Demyelinating Disease Program at Children’s Medical Center [ https://www.childrens.com/specialties-services/specialty-centers-and-programs/neurology/demyelinating-disease-program ]. Dr. Greenberg earned his medical degree at Baylor College of Medicine before completing an internal medicine internship at Chicago’s Rush Presbyterian-St. Luke’s Medical Center. He performed his neurology residency at the Johns Hopkins School of Medicine. He also holds an M.H.S. in molecular microbiology and immunology from the Bloomberg School of Public Health, as well as a bachelor’s degree in the history of medicine – both from Johns Hopkins. Prior to his recruitment to UT Southwestern in 2009, Dr. Greenberg was on the faculty of the Johns Hopkins Division of Neuroimmunology, serving as the Director of the Encephalitis Center and Co-Director of the nation’s first dedicated Transverse Myelitis Center. Dr. Greenberg splits his clinical time between adult and pediatric patients at William P. Clements Jr. and Zale Lipshy University Hospitals, Parkland, and Children’s Medical Center. His research focuses on better diagnosing, prognosticating, and treating demyelinating diseases and nervous system infections. He also coordinates clinical trials to evaluate new treatments to prevent neurologic damage and restore function to affected patients. 00:00 Welcome 01:44 Hosts and Guest Intro 05:06 Research Buckets Overview 07:55 Predicting Relapse Risk 11:46 Tregs and Immune Brakes 17:40 Attack Severity and Relapse 19:24 MOGAD Criteria Updates 21:38 Titers Explained Simply 26:32 Targeting MOG Antibodies 29:11 CAR T and Immune Reset 32:39 When Criteria Changes 33:52 Tolerance Research Boom 34:48 From Animals to Trials 37:17 Community Drives Progress 38:36 Meteoroid and cosMOG Clinical Trials 41:39 How These Drugs Work 44:02 FDA Approval and Access 45:49 Insurance Switch Concerns 48:39 Rituximab Dosing Debate 52:41 Why Antibodies Develop 54:18 Future Attack Patterns 55:47 CAR T Versus Rituximab 57:10 Lab Research and Support 01:00:51 Hope for a Cure 01:02:14 Closing and Resources

Ask the Expert 1403. Open Q&A on MOG Antibody Disease (MOGAD)

In this SRNA "Ask the Expert" episode moderated by Krissy Dilger, Dr. John Chen of the Mayo Clinic answered audience questions about MOG antibody disease (MOGAD). He discussed diagnosis and the importance of titers and live cell-based assays given possible false positives [00:02:42]. Dr. Chen reviewed acute management with early high-dose steroids, prolonged tapers, and escalation to plasma exchange for severe or steroid-refractory attacks, as well as evolving long-term options including IVIG/subcutaneous IG and IL-6 blockade [00:04:14]. Audience questions covered relapse prediction, vision recovery timelines, fatigue, pregnancy, heredity, symptom interpretation, and whether to stop immunotherapy when antibodies become undetectable [00:12:13]. Finally, Dr. Chen described current and upcoming research, including a trial that is currently enrolling participants, and future prospects for optic nerve regeneration while cautioning against unproven stem cell clinics [00:41:37]. John J. Chen, MD, PhD attended the University of Virginia for his undergraduate and combined MD/PhD degrees and completed his Ophthalmology residency and Neuro-Ophthalmology fellowship training at the University of Iowa. He then took a position at the Mayo Clinic in 2014 where he specializes in Neuro-Ophthalmology. Currently, he serves as a Consultant and Professor of Ophthalmology and Neurology, and Neuro-Ophthalmology Fellowship Director at the Mayo Clinic. Among Dr. Chen’s awards and honors are the AAO Senior Achievement Award, Top Doctors in Minnesota, the Heed Fellowship, Real World Ophthalmology Inspiring Academic Leader Award, Ophthalmology Teacher of the Year Award four times leading to induction to the Educators Hall of Fame, and the Mayo Clinic Distinguished Educator Award – awarded to the top educator at Mayo Clinic in Rochester. He is an Associate Editor for Ophthalmology and the Journal of Neuro-Ophthalmology, has authored more than 250 peer-reviewed publications, and focuses his research on ophthalmic imaging, idiopathic intracranial hypertension, and optic neuritis, particularly NMOSD and MOG antibody–associated disease. 00:00 Welcome and Introductions 01:08 What Is MOGAD? 02:42 Causes and Triggers 03:23 How MOGAD Is Diagnosed 04:14 Acute Attack Treatments 06:35 Steroid Side Effects 08:13 Testing During Treatment 09:09 Long Term Therapies 12:13 Interpreting MOG Positivity 16:51 Eye Symptoms and Vision Fluctuations 20:12 Antibody Titers and Severity 21:19 Relapse Risk After First Attack 23:09 Seizures and Encephalitis 24:17 Vision Recovery After Optic Neuritis 25:13 Acute Treatment Window 25:57 Hereditary Risk Questions 26:35 Stopping Azathioprine Safely 29:56 Managing Post Attack Pain 30:16 Steroids IVIG and Plasma Exchange 32:08 Infections as Triggers 33:01 Retesting MOG Antibodies 35:01 Fatigue and Workup 36:23 Prognosis and Life Expectancy 37:45 Tinnitus and Brain Pressure 39:05 Pediatric and Pregnancy Concerns 41:37 Trials and Future Regeneration 46:05 Research Resources and Wrap Up

Ask the Expert 1403. Open Q&A on MOG Antibody Disease (MOGAD)

In this SRNA "Ask the Expert" episode moderated by Krissy Dilger, Dr. John Chen of the Mayo Clinic answered audience questions about MOG antibody disease (MOGAD). He discussed diagnosis and the importance of titers and live cell-based assays given possible false positives [00:02:42]. Dr. Chen reviewed acute management with early high-dose steroids, prolonged tapers, and escalation to plasma exchange for severe or steroid-refractory attacks, as well as evolving long-term options including IVIG/subcutaneous IG and IL-6 blockade [00:04:14]. Audience questions covered relapse prediction, vision recovery timelines, fatigue, pregnancy, heredity, symptom interpretation, and whether to stop immunotherapy when antibodies become undetectable [00:12:13]. Finally, Dr. Chen described current and upcoming research, including a trial that is currently enrolling participants, and future prospects for optic nerve regeneration while cautioning against unproven stem cell clinics [00:41:37]. John J. Chen, MD, PhD attended the University of Virginia for his undergraduate and combined MD/PhD degrees and completed his Ophthalmology residency and Neuro-Ophthalmology fellowship training at the University of Iowa. He then took a position at the Mayo Clinic in 2014 where he specializes in Neuro-Ophthalmology. Currently, he serves as a Consultant and Professor of Ophthalmology and Neurology, and Neuro-Ophthalmology Fellowship Director at the Mayo Clinic. Among Dr. Chen’s awards and honors are the AAO Senior Achievement Award, Top Doctors in Minnesota, the Heed Fellowship, Real World Ophthalmology Inspiring Academic Leader Award, Ophthalmology Teacher of the Year Award four times leading to induction to the Educators Hall of Fame, and the Mayo Clinic Distinguished Educator Award – awarded to the top educator at Mayo Clinic in Rochester. He is an Associate Editor for Ophthalmology and the Journal of Neuro-Ophthalmology, has authored more than 250 peer-reviewed publications, and focuses his research on ophthalmic imaging, idiopathic intracranial hypertension, and optic neuritis, particularly NMOSD and MOG antibody–associated disease. 00:00 Welcome and Introductions 01:08 What Is MOGAD? 02:42 Causes and Triggers 03:23 How MOGAD Is Diagnosed 04:14 Acute Attack Treatments 06:35 Steroid Side Effects 08:13 Testing During Treatment 09:09 Long Term Therapies 12:13 Interpreting MOG Positivity 16:51 Eye Symptoms and Vision Fluctuations 20:12 Antibody Titers and Severity 21:19 Relapse Risk After First Attack 23:09 Seizures and Encephalitis 24:17 Vision Recovery After Optic Neuritis 25:13 Acute Treatment Window 25:57 Hereditary Risk Questions 26:35 Stopping Azathioprine Safely 29:56 Managing Post Attack Pain 30:16 Steroids IVIG and Plasma Exchange 32:08 Infections as Triggers 33:01 Retesting MOG Antibodies 35:01 Fatigue and Workup 36:23 Prognosis and Life Expectancy 37:45 Tinnitus and Brain Pressure 39:05 Pediatric and Pregnancy Concerns 41:37 Trials and Future Regeneration 46:05 Research Resources and Wrap Up