Neuro IGNITE Center
Neuro Innovative GeNetic Investigation and Treatment Evaluation Center
The Stanford Neuro IGNITE (Innovative GeNetic Investigation and Treatment Evaluation) Center is dedicated to improving the efficiency and volume of clinical trials for rare genetic neurological disorders. We are at the forefront of a new era in medicine, dedicated to accelerating the development and utilization of novel therapeutics for neurogenetic disorders. With decades of experience in identifying genetic causes and developing tailored trial designs, the Neuro IGNITE Center was developed to validate novel genetic treatments and then to address the complex challenges of residual neurological deficits post-treatment through the development and validation of innovative biomarkers and outcome measures. Our collaborative efforts with various Stanford programs and laboratories will increasingly strengthen our clinical trial designs and operational capabilities, ensuring we meet the growing demand to investigate new treatment modalities in this transformative era of neurogenetic medicine. The Neuro IGNITE Center will also integrate closely with existing programs at Stanford Health Center and Lucile Packard Children’s Hospital, emphasizing the importance of acquiring real-world data to shape the future of genetic treatment for neurological and neuromuscular disorders. Our mission is to bridge the gap between groundbreaking scientific discoveries and clinical application, ensuring that new treatments are developed and delivered to patients with unprecedented accuracy and efficiency. We achieve this by integrating structural and functional outcome measures with multiple tissue and circulating biomarkers, combined with innovative clinical trial designs to . Our work is about more than unidirectional clinical trials—but is focused on creating a self-correcting system where every patient's experience informs and improves the care for all.
Vision Statement: The Neuro IGNITE Center refines outcome measures, biomarkers and clinical trial design to optimize accurate and efficient investigation of novel therapeutic methods for neurologic and neuromuscular disorders.
Mission Statement: The Neuro IGNITE Center will promote, facilitate and support accelerated development and utilization of novel therapeutics for neurogenetic disorders: increase clinical trial operation and administration efficiency; improving the accuracy and reliability of outcome measures used for neurological disorders by collaborating with trialists, bioengineers and biomedical scientists; optimize clinical trial design, data collection and data analysis by collaborating with statisticians and bioinformatics specialists and integrating novel outcome measures and surrogate endpoints that accurately characterize treatment of genetic neurological disorders; efficiently integrate collection of post-approval real-world data to monitor treatment effects and determine new unmet needs.
Executive Committee
John W. Day, MD, PhD
Co-Director, and Medical Director, Neuro IGNITE
Professor of Neurology, Pediatrics (Genetics), and by courtesy, Pathology
Director, Division of Neuromuscular Medicine
Dr. John W. Day is a distinguished figure in neuromuscular medicine and a Co-Director of the Neuro IGNITE Center, bringing more than 30 years of experience to the program. As a Professor of Neurology, Pediatrics (Genetics), and Pathology, and the Director of the Division of Neuromuscular Medicine at Stanford University, he is a driving force behind the center's mission to bridge the gap between genetic discovery and clinical application.
Dr. Day's expertise in translational science is foundational to the Neuro IGNITE Center's innovative approach. His extensive experience includes investigating the genetic causes and multisystemic effects of neuromuscular disorders, as well as designing and directing groundbreaking clinical trials. He has been at the forefront of evaluating novel therapeutics, including antisense oligonucleotides and gene replacement therapies, directly aligning with the center's focus on genetic investigation and treatment evaluation.
Before establishing a comprehensive Division of Neuromuscular Medicine at Stanford in 2011, Dr. Day founded and directed the Paul and Sheila Wellstone Muscular Dystrophy Center at the University of Minnesota. This history of creating and leading impactful, multidisciplinary programs demonstrates his profound commitment to accelerating scientific discovery and advancing patient care through collaborative innovation, which is the core of the Neuro IGNITE Center.
Tina Duong, MPT, PhD
Co-Director, Neuro IGNITE
Senior Research Scientist
Director, Clinical Outcomes Research & Development, Division of Neuromuscular Medicine, Stanford University
Assistant Professor, Department of Medicine and Rehabilitation, University of California, Davis
Dr. Tina Duong, a leader in translational science and drug development, serves as the Co-Director of the Neuro IGNITE Center and Director of Clinical Outcomes Research and Development at Stanford. With over 20 years of clinical and trial experience, she brings a deep understanding of neuromuscular disease, having dedicated her career to evaluating, treating, and supporting patients.
Her expertise is pivotal to the Neuro IGNITE Center's mission, where she is a driving force behind initiatives in novel trial designs and the development of innovative outcome measures. Her work focuses on accelerating the drug development landscape by integrating technology and digital biomarkers into clinical outcomes and evaluations. This approach provides a complementary, data-rich perspective to traditional performance-based measures, enabling a more comprehensive understanding of disease progression and addressing gaps in measuring therapeutic response.
Prior to her arrival at Stanford in 2015, Dr. Duong’s work at the Cooperative International Neuromuscular Research Group (CINRG) provided a valuable foundation in clinical trial readiness, and design of natural history studies. This experience was essential in supporting drug approvals for diseases like Duchenne muscular dystrophy and Spinal Muscular Atrophy (SMA). The insights gained from the SMA experience, in particular, helped inform the importance of collecting and interpreting real-world data, a crucial step for continued expansion of post-marketing drug information, informing future drug development and approval.These lessons are directly integrated into the Neuro IGNITE Center’s mission to deepen and streamline novel therapeutic clinical development and accelerate patient access and safe use of new therapies.
Dr. Duong is committed to fostering multidisciplinary collaborations, bringing together her clinical insight and technological foresight. Her work embraces the "Learn From Every Patient" model, an approach where unique insights from each individual's journey help inform rare disease research. This work supports the necessary translational research from early exploratory studies (Phase 0) to clinical care, helping to streamline the development of more targeted therapies and, ultimately, improve patient access to treatment.
Shirley Paulose, MBBS, MS
Executive Manager, Neuro IGNITE
Clinical Research Manager
Shirley Paulose brings over two decades of clinical research expertise to the Stanford Neuromuscular Research Program, where she has been a key member since 2006. As the Executive Manager of the Neuro IGNITE Center and as the Senior Clinical Research Manager of the Neuromuscular Research Program, she provides strategic leadership for the program’s clinical trials ensuring the highest standards of quality, accuracy, and regulatory compliance in every step of study execution. Her ability to align priorities and build cross-functional relationships enables her to provide effective operational oversight to the program.
Shirley’s clinical experience as an Ophthalmologist with additional experience in Pediatrics, and her strong foundation in clinical research helps her to navigate the complexities of the clinical trials on rare diseases with precision, efficiency and compassion. This multifaceted experience uniquely positions her to be an efficient and knowledgeable leader.
She is equally committed to mentorship, investing in the professional growth of researchers and investigators by instilling best practices, operational rigor, and patient-centered treatment programs. With her dedication, expertise and collaborative spirit, she plays a pivotal role in driving innovation and excellence in neuromuscular research and patient care.
Diana H. Tran, MPH
Project Management Facilitator, Neuro-IGNITE
Diana serves as a Project Management Facilitator for the Neuro IGNITE Center and the Stanford Neuromuscular Division, where she leads project coordination and supports interdisciplinary collaboration to advance research initiatives. She began her journey with the Neuromuscular Division in February 2022 as a Research Administrator. Diana earned her Master of Public Health (MPH) from California Baptist University in 2021. Her professional background spans the non-profit, healthcare, and academic sectors, providing her with a well-rounded perspective on program development and community impact. Prior to joining Stanford, she designed and oversaw academic and athletic programs for at-risk youth—work that highlights her deep commitment to health equity, community empowerment, and service.
Advancement, Community, and Engagement
Jacinda Sampson, MD, PhD
Clinical Professor
Adult Neurology
Maxwell A Greene, MD
Clinical Assistant Professor
Adult Neurology
Clinical Research Advisory Committee (CRAC)
Srikanth Muppidi, MD
Clinical Professor
Adult Neurology
Neelam Goyal, MD
Clinical Professor
Adult Neurology
Carolina Tesi Rocha, MD
Clinical Professor
Pediatric Neurology
Clinical Professor (By Courtesy)
Pediatrics
Maxwell A Greene, MD
Clinical Assistant Professor
Adult Neurology
Jenna Klotz, MD
Clinical Associate Professor
Pediatric Neurology
Clinical Associate Professor, by courtesy
Pediatrics
Scientific Advisory Committee (SAC)
Carolina Tesi Rocha, MD
Clinical Professor
Pediatric Neurology
Clinical Professor (By Courtesy)
Pediatrics
Jacinda Sampson, MD, PhD
Clinical Professor
Adult Neurology
Matthew Wheeler, MD, PhD
Associate Professor of Medicine
Cardiovascular Medicine
Clinical Trial Advisory Committee (CTAC)
Michael Hagerman, MBA
Cofounder/CEO DirectorsEdge Inc.
Advisor, Christopher Project
(External biomedical research advisor)
Sharon Sha, MD, MS
Clinical Professor
Adult Neurology
Carolina Tesi Rocha, MD
Clinical Professor
Pediatric Neurology
Clinical Professor (By Courtesy)
Pediatrics
Administrative Board of Advisors
Michael Hagerman, MBA
Cofounder/CEO DirectorsEdge Inc.
Advisor, Christopher Project
(External biomedical research advisor)
Ruth O'Hara, PhD
Director, Stanford’s Center for Clinical & Translational Education and Research (Spectrum),
Senior Associate Dean of Research, Lowell W. and Josephine Q. Berry Professor
Scott L. Delp, PhD
Director, Wu Tsai Human Performance Alliance at Stanford
James H. Clark Professor, School of Engineering
Professor of Bioengineering, of Mechanical Engineering, and by courtesy, of Orthopaedic Surgery
Joseph C. Wu, MD, PhD
Director, Stanford Cardiovascular Institute
Simon H. Stertzer, MD, Professor of Medicine and Radiology
Euan A. Ashley, MD, PhD
Roger and Joelle Burnell Professor of Genomics and Precision Health
Arther L. Bloomfield Professor of Medicine and Professor of Genetics, of Biomedical Data Science and, by coutrsy, of Pathology, Chairman, Department of Medicine
Matthew Porteus, MD, PhD
Sutardja Chuk Professor of Definitive and Curative Medicine
Neuromuscular Fellows
Julia Greenberg, MD
Trev (Martavius) Lovall, MD, MS
Manny (Emanuel) Mostofi, MD
Muhannad Seyam, MD
Neuromuscular Research Staff
Constance de Monts PT, DPT
Research Physical Therapist
Sally Dunaway Young, PT, DPT
Research Physical Therapist
Richard Gee, PT
Research Physical Therapist
Stanford Children's Health
Lisa Ghiglieri
Life Science Research Professional
Rachana Patil
Life Science Research Professional 2
Melina Rogers
Research Data Analyst
Audrey Chun
Research Data Scientist
Tahereh Kamali, PhD
Instructor
Bradley Hamilton, PhD
Instructor
Nathan Hageman
Research Scientist
Marwa Zafarullah, PhD
Postdoctoral Research Fellow
Tia Lum
Clinical Research Manager 1
Veronica Schnyer
Clinical Research Manager 1
Lesly Welsh, CCRC, CMA
Clinical Research Manager 1
Rabia Farooquee
Clinical Research Coordinator 2
Habib Mofakham Fini
Clinical Research Coordinator 2
Susan Thomas
Clinical Research Coordinator 2
Lidia Choniawko
Clinical Research Coordinator 2
Wendy Ao
Clinical Research Coordinator Associate
Raiye Hailu
Clinical Research Coordinator Associate
Sarah Ismail
Clinical Research Coordinator Associate
Lin Karman
Clinical Research Coordinator Associate
Sabrina Salvatore
Clinical Research Coordinator Associate
Natalia Wieczorek
Clinical Research Coordinator Associate
Shelby Smith
Clinical Research Coordinator Associate
Meng Anna Yao
Clinical Research Coordinator Associate
Sonia Pathak, MA
Clinical Research Coordinator Associate
Mahi Gandhi, MS
Clinical Research Coordinator Associate
Libby Yatsu
Clinical Research Coordinator Associate
Michelle Jepsen
Clinical Research Coordinator Associate
Nikita Sehgal, MS
Clinical Research Coordinator Associate
Dong T. Nguyen, MA
Administrative Associate 2
Translational Science
From the Bench to the Bedside: Our Integrated Approach
The Neuro IGNITE Center transforms basic and translational discoveries into tangible patient benefits. Our integrated cores work in synergy to push the boundaries of what is possible in neurogenetic treatment.
Biomarker Discovery: We are at the forefront of identifying and validating biomarkers that provide a deeper understanding of disease. We utilize machine learning and artificial intelligence to define novel biomarkers from a multimodal approach that integrates circulating, imaging, and functional digital health outcomes. Our comprehensive view of disease progression and therapeutic response is created by integrating clinical data with insights from blood and CSF proteomic and other circulating biomarkers, muscle biopsies, autopsies and other patient-derived specimens, and advanced PET, MRI and ultrasound imaging. The center’s repository of clinically characterized specimens is an essential resource for both basic and translational investigations.
Outcomes & Innovation: Our work focuses on refining how we measure what matters to patients. We do this by developing and adapting novel outcome measures, including digital and patient-reported outcomes.Collaborating with experts like bioengineers, we integrate wearables and other digital tools into clinical trials to provide a more precise and comprehensive picture of a patient’s health. This approach allows us to accurately monitor structural and functional characteristics, improving the sensitivity for detecting important changes in different neuromuscular diseases. By defining the Minimal Clinically Important Difference (MCID), we ensure that statistical improvements truly translate into meaningful clinical benefits for patients.
Trial Design & Readiness: Our expertise lies in designing trials for rare and ultra-rare disorders.We develop tailored trial designs and utilize novel methods of data acquisition and analysis to achieve sufficient efficacy even with small, heterogeneous patient populations.
This work is supported by a robust operational infrastructure at the Neuro IGNITE Center, which serves as a platform for quickly translating research into diverse patient populations, including first-in-human studies Our data management team’s close collaboration with biostatisticians and bioinformatics specialists streamline data collection, and organization, supporting research and and clinical care programs.
Artificial Intelligence & Machine Learning for Therapeutic Response Evaluation in Neuromuscular Disorders
We develop AI and machine learning solutions to enhance the evaluation of therapeutic response across neuromuscular disorders. Our models integrate multimodal data, including neuroimaging, electrophysiology, molecular profiles, neurocognitive data, and patient-reported outcomes, to quantify treatment effects and inform individualized care. In alignment with genetic interventions, our AI/ML work supports data-driven decision-making in both clinical trials and real-world applications. We are developing scalable foundational models capable of learning from heterogeneous, multisite datasets. These models are designed to capture both shared and disease-specific patterns across neuromuscular conditions, enabling cross-disorder insights and flexible deployment. By combining deep learning with interpretable outputs and clinically meaningful outcome measures, we aim to create practical tools that support therapeutic evaluation and bring advanced analytics closer to everyday clinical and research workflows.
The Neuro IGNITE Center is a hub of active research studies and collaborations that are advancing the field of neuromuscular medicine. Our projects span the entire translational spectrum, from natural history studies to sponsored clinical trials for gene therapies.
Clinical Trials & Studies: We lead and participate in a wide range of studies, including sponsored studies, investigator-initiated studies, and natural history projects. This includes groundbreaking work in antisense oligonucleotide and AAV gene therapy treatments.
Our neuromuscular research team has collaborated in the testing and development of many antisense oligonucleotide (ASO) and small interfering RNA (siRNA) compounds for neuromuscular conditions including spinal muscular atrophy (SMA), myotonic dystrophy (DM), amyotrophic lateral sclerosis (ALS), and facioscapulohumeral muscular dystrophy (FSHD). These ASO and siRNA approaches typically address the root cause of neuromuscular diseases, either by reducing production of unhealthy molecules or increasing production of missing healthy molecules. Our team’s greatest success with ASO treatments to date has been with the clinical trial of nusinersen to treat SMA. We delivered the first in-human treatment to an infant with SMA in 2013, and assisted in multiple clinical trials of nusinersen which led to the rapid FDA approval in 2016. Now more than 14,000 people with SMA receive nusinersen treatment worldwide, and the Neuro IGNITE center continues to collect real-world data from patients on nusinersen treatment as the disease course changes in response to treatment, and develop and adapt new approaches to address the remaining symptoms of SMA. Experience with nusinersen established the role of antisense oligonucleotides in treating neurological disorders. We also played significant roles in validating risdiplam, an orally administered splicing modifier that the FDA approved for SMA, and onasemnogene abeparvovec, the first AAV gene therapy approved by the FDA for systemic infusion.
Real-World Data: We efficiently integrate real-world clinical data to monitor the long-term effects of treatments and identify new unmet needs. This continuous loop of data collection and analysis drives quality improvement and helps us define toxicities, optimal management, and new phenotypes emerging in patients receiving genetic treatments.
A Changing Landscape: The advent of disease-modifying treatments has created a need for precision medicine and novel neuroregenerative methods to address residual deficits. The Neuro IGNITE Center is positioned to develop biomarkers, outcome measures, and trial designs required to validate these complementary treatments to improve the quality of life for all neuromuscular patients, not just those in a single trial.
The Stanford Neuro IGNITE Center is at the forefront of innovation in genetic neurological disorders, fostering collaborations both within Stanford and with external partners to transform clinical trial methodologies and enhance therapeutic development, all while maintaining a strong focus on patient-centered care.
Our dedication to advancing the field of neurogenetic medicine is further strengthened by a diverse array of esteemed collaborators, including:
Stanford Collaborators
Stanford Neurology & Neurological Sciences
The Stanford Neurology & Neurological Sciences Department is a recognized center of excellence in neurology for over four decades. With over 140 faculty members and access to four premier teaching hospitals, the department is committed to advancing patient care through cutting-edge treatments and innovative research.
Research at Stanford Bioengineering seeks to measure and understand the world around us with utmost care and precision, to recreate the phenomena we witness, and to design tools and approaches with real applications that impact humanity.
The Human Performance Laboratory
The Human Performance Laboratory is a center for excellence in health science providing a unique link between Stanford’s prestigious Athletics program, the School of Medicine, and various Academic Departments within the University. The center performs interdisciplinary research integrating biomechanics, biomedical engineering, physiology, psychology, and rehabilitation.
Wu Tsai Neuroscience Institute
The Wu Tsai Neurosciences Institute is dedicated to understanding how the brain gives rise to mental life and behavior, in health and disease.
The Center for Definitive and Curative Medicine
In the Stanford Center for Definitive and Curative Medicine (CDCM), researchers are use cell and gene therapies to treat diseases from common diagnoses such as diabetes to rare diseases of the brain, blood, skin, immune system, and other organs. The CDCM translates exploratory and preclinical studies into definitive cures for currently incurable diseases.
Established in 2004, the Stanford Cardiovascular Institute establishes educational and training programs in cardiovascular medicine and promotes research and clinical applications through awarded seed grants.
The Innovative Medicines Accelerator
The Stanford Innovative Medicines Accelerator (IMA) facilitates the translation of Stanford research discoveries into new medicines while expanding knowledge of human biology.
The Lucas Center for MRI Research
The Richard M. Lucas Center for Imaging houses resources devoted to research in magnetic resonance imaging (MRI), spectroscopy (MRS), and X-Ray/CT imaging supports interdisciplinary research illuminating human physiology and the diagnosis and treatment of human diseases. It advances imaging in healthcare at Stanford through technology creation and development, translational research and education.
Institute for Stem Cell Biology and Regenerative Medicine
The Stanford Institute for Stem Cell Biology and Regenerative Medicine was established in 2002 to build on Stanford’s leadership in stem cell science and to set the foundations for regenerative medicine.The institute is devoted to exploring stem cell generation, differentiation.and therapeutic potential.
The Brain Organogenesis Program
The Stanford Brain Organogenesis Program brings together expertise in neurosciences, stem cell biology, engineering, chemistry, medicine and law/ethics to non-invasively construct and deconstruct inaccessible stages of human brain development and function in health and brain disorders.
The Center for Cognitive and Neurobiological Imaging
The Stanford Center for Cognitive and Neurobiological Imaging (CNI) is a shared facility, that provides resources for researchers and students in cognitive and neurobiological sciences.
Affiliated Partners
Academic Institutions
Columbia University
University of California, Irvine
University of California, San Diego
University of Florida
University of Kansas Medical Center (KUMC)
University of Miami
University of Rochester
Virginia Commonwealth University
Industry Partners
Annji Pharmaceutical
Abcuro, Inc.
Alcyone Therapeutics
Alexion Pharmaceuticals
AMO Pharma
Arcellx, Inc.
Argenx, Inc.
Astellas Pharma
Atyr Pharma
Avidity Biosciences
Biogen, Inc.
Bristol Meyer Squibb
Catalyst Pharma
Cytokinetics, Inc.
Dyne Therapeutics
Edgewise Therapeutics
Expansion Therapeutics
Glaxo SmithKline
Insmed Pharmaceuticals
Ionis Pharmaceuticals
Jacobus Pharma
Janssen (now Johnson & Johnson Innovative Medicine)
Kyverna Therapeutics
Mallinckrodt
NMD Pharma
Novartis Gene Therapies
Perkin Elmer
PepGen, Inc.
Pfizer, Inc.
PTC Therapeutics
Regenxbio, Inc.
RESTEM, LLC
Roche / Genentech
Sarepta Therapeutics
Sanofi Pharmaceuticals
Satellos Bioscience
Scholar Rock, Inc.
Spur Therapeutics
Takeda Pharmaceuticals
Vertex Pharmaceuticals
Viking Therapeutics
Community, Non-Profit Partners, & Clinical Research Networks
A Foundation Building Strength for Nemaline Myopathy
CMT Association
Cure SMA
FSHD-CRN
Inherited Neuropathies Consortium
Jain Foundation
Muscular Dystrophy Association
Myotonic Dystrophy Foundation
SMA Foundation
Solve FSHD
Solve GNE
The Marigold Foundation
The Myotonic Dystrophy Clinical Research Network (DMCRN)
Wilson-5 Foundation
Government Agencies
Department of Defense
U.S. Food and Drug Administration (FDA)
National Institutes of Health (NIH)