NIH-funded Phase 2B clinical trial for NMDAR encephalitis treatment continues to successfully enroll patients at dozens of sites, including WashU Medicine

The ExTINGUISH NMDAR Encephalitis Trial

An ongoing clinical trial designed to answer critical questions regarding treatment protocols for anti-NMDA receptor encephalitis, a relatively rare but often devastating condition, is continuing to expand trial sites and enroll participants, building excitement in the medical and patient communities. At Washington University School of Medicine in St. Louis, David Clifford, MD, the Melba and […]

Understanding the genetic factors behind pediatric pilocytic astrocytoma and possible treatments

White coat with WashU Medicine logo

Pediatric pilocytic astrocytoma (PA) is a common type of low-grade brain tumor in children, influenced by specific genetic changes. Researchers at Washington University School of Medicine in St. Louis have conducted a study, published in Genes & Development, using advanced stem cell techniques to investigate the genetic alterations that cause PA and how they affect […]

Promising risk assessment results for new pediatric stroke treatment option

patient with IV

A recent safety surveillance paper published in Neurology reported that of eleven pediatric patients who were treated with tenecteplase for suspected stroke, there were no instances of intracranial bleeding or other safety concerns. This study is an important first step toward further research to establish the safety and efficacy of tenecteplase for treating pediatric stroke. […]

Novel Gene Therapy Clinical Trial Targets Rett Syndrome

Gloved hands hold syringe to glass vial

Washington University School of Medicine in St. Louis is one of only ten sites in the world participating in a pioneering clinical trial evaluating TSHA-102, an investigational gene therapy for the treatment of Rett syndrome, a severe neurodevelopmental disorder primarily affecting girls. The REVEAL Phase 1/2 clinical trial represents a significant advancement in the quest […]

Researchers define new subtypes of common brain disorder (Links to an external site)

An MRI scan shows a Chiari type-1 malformation, in which the cerebellum extends beyond the gap in the skull where it connects to the spinal cord. Researchers at Washington University School of Medicine in St. Louis have used AI tools to describe three sub-types of Chiari type-1, which will help guide clinicians to make the most effective treatment decisions for their patients.

Roughly 4% of the population is affected by a congenital brain malformation that has eluded researchers’ efforts to find causes and treatments. For the condition, Chiari type-1 malformation, the diagnosis is straightforward: the lower part of the brain, known as the cerebellum, protrudes at least five millimeters through the gap in the skull that connects […]

Geisler, McPherson awarded grants as part of ICTS’s Clinical and Translational Research Funding Program

Stefanie Geisler and Laura McPherson

Washington University Institute of Clinical and Translational Sciences (ICTS) and The Foundation for Barnes-Jewish Hospital have awarded Stefanie Geisler, MD, and Laura McPherson, PT, DPT, PhD, grants as part of the 17th annual Clinical and Translational Research Funding Program (CTRFP). This program is the largest internal grant funding program in the ICTS, requiring applicants to […]

Washington University Neurofibromatosis Center Celebrates 30 Years of NF

Angie Hirbe adult NF care

One of the largest and most comprehensive centers in the world specializing in the care of people with Neurofibromatosis (NF) and related disorders — the Washington University NF Center — is celebrating 30 years of NF. Neurofibromatosis (NF) is a set of four rare genetic medical conditions with which children and adults are prone to […]

Tuberous Sclerosis Center at Washington University School of Medicine in St. Louis expands with addition of Adult TSC Director Nascimento

Fábio A. Nascimento, MD, and Patty Schaefer examine a patient

The Washington University Tuberous Sclerosis Center is recognized by the Tuberous Sclerosis Complex Alliance as one of only 13 TSC Centers of Excellence across the globe and the only in Missouri. Its multispecialty team is equipped to provide the highest level of comprehensive, medical care for children and adults with tuberous sclerosis complex while conducting cutting-edge […]

Publication from Horie, Sato, Ghoshal and Bateman wins ISTAART 2023 Publication of the Year Award

Horie, Sato, Ghoshal, Bateman

An article published in Nature Medicine from neurologists Kanta Horie, PhD, Chihiro Sato, PhD, Nupur Ghoshal, MD, PhD, Randall Bateman, MD, and colleagues has won the International Society to Advance Alzheimer’s Research and Treatment (ISTAART) 2023 Publication of the Year Award. The article is titled, “CSF tau microtubule-binding region identifies pathological changes in primary tauopathies.” […]