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Neurosciences

Early Temporization Linked to Better Early Development in Premature Infants With Posthemorrhagic Hydrocephalus
 Early Temporization Linked to Better Early Development in Premature Infants With Posthemorrhagic Hydrocephalus 864 576 Nationwide Children's
By: Nationwide Children's
Neurosurgeon Albert Isaacs posed in the operating room, looking at the camera.

A new HCRN study led by Nationwide Children’s links early temporary cerebral spinal fluid diversion and timely shunt conversion to better early development in preterm infants with posthemorrhagic hydrocephalus. A new Hydrocephalus Clinical Research Network (HCRN) study, published in Journal of Neurosurgery: Pediatrics, suggests that starting with temporary cerebrospinal fluid (CSF) diversion and converting to…

One Gene, Big Impact: The Science Behind a New Therapy for Brain and Muscle Health
 One Gene, Big Impact: The Science Behind a New Therapy for Brain and Muscle Health 1024 683 Madison Storm
By: Madison Storm
Dr. Zarife Sahenk, MD, PhD

A promising new gene therapy developed at Nationwide Children’s Hospital is offering hope for new patients with neurodegenerative and muscle disorders. Led by Zarife Sahenk, MD, PhD, a neurologist, principal investigator in the Jerry R. Mendell Center for Gene Therapy and director of Clinical and Experimental Neuromuscular Pathology at the Abigail Wexner Research Institute, the…

Nationwide Children’s Hospital Completes One of the World’s First Prenatal Treatments for Spinal Muscular Atrophy
 Nationwide Children’s Hospital Completes One of the World’s First Prenatal Treatments for Spinal Muscular Atrophy 1024 600 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Baby Bryson laughing at a check up following treatment for SMA.

Investigational third-trimester, transplacental “bridging” therapy with risdiplam — followed by rapid postnatal treatment — aims to protect motor neurons during a critical window before and immediately after birth.  When Stacy Auker found out she was pregnant with her third child, she knew a diagnosis of spinal muscular atrophy (SMA) was a possibility.  “We have relatives who have been affected, so I…

Study Shows Younger Children Experience Persistent Symptoms Following Concussion
 Study Shows Younger Children Experience Persistent Symptoms Following Concussion 1024 540 Abbie Miller and Katelyn Scott
By: Abbie Miller and Katelyn Scott

Nearly 30% of children younger than 6 years who had a concussion during the study had prolonged symptoms, which can affect learning and behavior later in childhood. When people typically think of concussion, the first type of patient that comes to mind is a youth athlete. However, concussion is also common in early childhood, due…

Behavioral Intervention Program for Children With Autism Spectrum Disorder Undergoing Epilepsy Evaluation: Increasing Phase 1 Monitoring Rates
 Behavioral Intervention Program for Children With Autism Spectrum Disorder Undergoing Epilepsy Evaluation: Increasing Phase 1 Monitoring Rates 1024 683 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

A behavioral intervention program that started as a quality improvement project evolved to a new standard of care — increasing access and compliance with Phase 1 monitoring for children with intractable epilepsy and autism spectrum disorder. A recent study led by Mary Wojnaroski, PhD, a psychologist at Nationwide Children’s Hospital, reported a sustained, multi-year increase…

Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease
 Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease 1024 576 Abbie Miller
By: Abbie Miller

Researchers at Nationwide Children’s Hospital recently administered a novel gene therapy targeting SLC6A1, advancing precision medicine for children who previously could not be treated. Maxwell, age 8, made history this fall, when he received a bespoke gene therapy targeting his ultrarare disease. This wasn’t the first time he worked with the team at Nationwide Children’s…

Expanding the Evidence for Cannabidiol in Focal Epilepsy
 Expanding the Evidence for Cannabidiol in Focal Epilepsy 1024 575 Pam Georgiana
By: Pam Georgiana
Young boy turned sideways in front of a dark background, with dark lighting and an illustration of his brain appearing in front of the side of his head

A multicenter study led by Nationwide Children’s Hospital demonstrates long-term efficacy and safety of Epidiolex® for treatment-resistant seizures. As global use of cannabidiol for seizure management increases, most research has targeted generalized epilepsy. This leaves a major evidence gap for patients with focal seizures and epilepsy. Anup D. Patel, MD, FAAN, FAES, FCNS, neurologist and…

Meet Our Expert: Albert Isaacs, MD, PhD, Neurosurgeon and Genomic Scientist
 Meet Our Expert: Albert Isaacs, MD, PhD, Neurosurgeon and Genomic Scientist 864 576 Lauren Dembeck
By: Lauren Dembeck
Neurosurgeon Albert Isaacs posed in the operating room, looking at the camera.

Albert Isaacs, MD, PhD, is a pediatric neurosurgeon at Nationwide Children’s Hospital and an assistant professor of Neurological Surgery at The Ohio State University College of Medicine. His research focuses on uncovering the molecular and immunologic drivers of neonatal hydrocephalus, with the goal of developing preventative treatments for at-risk infants. Dr. Isaacs’ extensive background and…

Featured Researcher – Eric Sribnick
 Featured Researcher – Eric Sribnick 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

When children experience a traumatic brain injury (TBI) as well as significant injuries elsewhere in the body—such as a liver or spleen laceration or a broken bone—they face a substantially increased risk of subsequent infection. Research suggests this may stem from a sort of shock to the immune system leading to immunoparalysis, which leaves children…

Improving Pediatric Clinical Trials by Incorporating Parent Input
 Improving Pediatric Clinical Trials by Incorporating Parent Input 1024 681 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

A Parent Council in an ongoing pediatric stroke rehabilitation trial provides important insights into how parent involvement can inform and enrich the research process. While the need for diverse and inclusive clinical trial populations has been acknowledged for the last few decades, the value of input into trial design from the patient (and in pediatrics,…

A Novel EEG Evaluation Method for Children With Infantile Spasms
 A Novel EEG Evaluation Method for Children With Infantile Spasms 1024 677 Lauren Dembeck
By: Lauren Dembeck

Clinician-researchers have developed a rapid training program to learn the BASED score, a novel EEG evaluation method. The easily accessible training program allows clinicians to reliably determine the response to treatment. Infantile epileptic spasms syndrome is a severe form of early childhood epilepsy. Onset typically occurs in infants 4 to 8 months old and is…

Selective Dorsal Rhizotomy for Cerebral Palsy: Significant Gains in Mobility and Reduction in Spasticity Treatments
 Selective Dorsal Rhizotomy for Cerebral Palsy: Significant Gains in Mobility and Reduction in Spasticity Treatments 928 1024 Erin Gregory
By: Erin Gregory

Recent research shows that selective dorsal rhizotomy (SDR) significantly boosts motor function in children with cerebral palsy (CP), particularly those who are ambulatory. The study highlights motor improvements and a reduced need for spasticity treatments, emphasizing SDR’s potential to enhance outcomes and quality of life for children with CP. Key Findings: Improvements in Motor Function…

Optimizing Outpatient Epilepsy Care With Digital Seizure Action Plans
 Optimizing Outpatient Epilepsy Care With Digital Seizure Action Plans 1024 683 Pam Georgiana
By: Pam Georgiana

Customized seizure action plans enhance care and family use. Many schools and caregivers require families of children with epilepsy to provide a written seizure action plan (SAP) signed by their primary care provider. The Epilepsy Foundation provides a printable PDF form to fill out, print and sign. However, the form is limited in providing space…

Featured Researcher – Christopher Bartlett, PhD
 Featured Researcher – Christopher Bartlett, PhD 150 150 Alaina Doklovic
By: Alaina Doklovic

Throughout Dr. Bartlett’s career, he has dedicated his time to the development of innovative statistical methods to enhance our understanding of childhood development, from pre-term birth to young adulthood. Specializing in the design and application of data analytic paradigms, his work significantly contributes to elucidating the complex interplay between environmental factors and childhood intrinsic characteristics.…

4 Questions With Jeffrey Leonard, MD
 4 Questions With Jeffrey Leonard, MD 1024 683 Abbie Miller
By: Abbie Miller

Jeffrey Leonard, MD, is chief of Neurosurgery and the Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neurosurgery at Nationwide Children’s Hospital. He is also a principal investigator in the Center for Childhood Cancer Research in the Abigail Wexner Research Institute and a professor of Neurological Surgery at The Ohio State University College…

Accelerating Advances in Epilepsy Surgery
 Accelerating Advances in Epilepsy Surgery 1024 683 Abbie Miller
By: Abbie Miller

One program is bringing together clinical care, surgery and research to create a brighter future for children with intractable epilepsy. Childhood-onset epilepsy affects 1% of children worldwide. Up to a third of patients with epilepsy will have medically refractory epilepsy, continuing to have seizures despite using two or more antiseizure medications. Options for this group…

Neurorehabilitation for Children With Batten Disease
 Neurorehabilitation for Children With Batten Disease 1024 683 Lauren Dembeck
By: Lauren Dembeck

Therapy should be tailored to each child’s needs. Batten disease comprises a rare group of related genetic disorders characterized by progressive neurodegeneration. The disease primarily presents in childhood as seizures, vision loss, and developmental regression. Neurorehabilitation services, including physical therapy (PT), occupational therapy (OT), and speech-language pathology therapy (SLP), can delay loss of function and…

Meet Toni Pearson, MD: Pediatric Movement Disorders Expert
 Meet Toni Pearson, MD: Pediatric Movement Disorders Expert 150 150 Pam Georgiana
By: Pam Georgiana

Toni S. Pearson, MD, joined Nationwide Children’s in 2022 as an attending pediatric neurologist and professor of pediatrics at The Ohio State University College of Medicine. She specializes in childhood movement disorders, such as dystonia, chorea, parkinsonism, ataxia, myoclonus, tremors, tics, and spasticity. Dr. Pearson is also developing a fellowship training program for a new…

Kids With Concussions Need Specialized Assessment and Care
 Kids With Concussions Need Specialized Assessment and Care 1024 683 Sean Rose, MD
By: Sean Rose, MD

Learn why cutting-edge care and research specifically for children matters. The 2020 National Health Interview Survey reported that 6.8% of children experience symptoms of a concussion or brain injury in their lifetime. However, only 3.9% had received a diagnosis or treatment of a concussion or brain injury from a health care provider. The Concussion Center…

Outcomes After Surgical Interventions for Abusive Head Trauma
 Outcomes After Surgical Interventions for Abusive Head Trauma 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Study suggests that most children can make clinically meaningful recoveries after neurosurgical intervention. Abusive head trauma (AHT) is a leading cause of injury and death in children under 5 years, according to the Centers for Disease Control and Prevention (CDC), yet the scientific literature on neurosurgical intervention for this population is mixed. Given the range…

Novel Cause of Brain Mosaicism and Focal Epilepsy Identified
 Novel Cause of Brain Mosaicism and Focal Epilepsy Identified 1024 575 Abbie Miller
By: Abbie Miller

In Nature Genetics, researchers report a novel mechanism for the origin of brain chromosomal mosaicism and link brain mosaic chromosome 1q gain to a distinct clinical phenotype.   In most people, every cell in their body contains the same genetic information. However, sometimes people can have two or more genetically different sets of cells. This…

Does Etiology Impact Outcomes After Hemispherectomy?
 Does Etiology Impact Outcomes After Hemispherectomy? 1024 575 Jessica Nye, PhD
By: Jessica Nye, PhD

Patients with cerebral palsy (CP) who have comorbid medically intractable epilepsy (MIE) had similar quantitative functional and seizure outcomes following functional hemispherotomy (FH) or anatomic hemispherectomy (AH), regardless of vascular or dysplastic MIE etiologies.   Early onset seizures are a common comorbidity in CP, affecting between 30% and 50% of patients. Among the patients with…

Deep Brain Stimulation Ameliorates Symptoms of Genetic Movement Disorder
 Deep Brain Stimulation Ameliorates Symptoms of Genetic Movement Disorder 150 150 Mary Bates, PhD
By: Mary Bates, PhD

First report of DBS for management of tremor in a treatment-resistant AIFM1-related disorder.   In a new case series, researchers from Nationwide Children’s report using deep brain stimulation (DBS) to help manage tremor due to a genetic movement disorder in two brothers. The report, published in the journal Pediatric Neurology, suggests DBS may be useful…

Understanding the Cellular Mechanisms to Drive New Therapies for Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech and Seizures (NEDAMSS)
 Understanding the Cellular Mechanisms to Drive New Therapies for Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech and Seizures (NEDAMSS) 1024 577 Jessica Nye, PhD
By: Jessica Nye, PhD

Derived cells from patients with NEDAMSS exhibit perturbed cellular respiration and poor neuronal survival, both of which can be improved with CuATSM treatment. NEDAMSS (neurodevelopmental disorder with regression, abnormal movements, loss of speech, and seizures) is a rare neurological disorder discovered in 2018 with only 25 formally diagnosed patients worldwide. It is primarily caused by…

Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy
 Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy 1024 575 Lauren Dembeck
By: Lauren Dembeck

Duchenne muscular dystrophy (DMD) is a devastating progressive muscle-wasting disease that leads to loss of motor and cardiorespiratory function. The disease is caused by mutations in the DMD gene that result in the loss of expression of the dystrophin protein, which plays a critical role during contraction and relaxation of muscle. To date, the U.S.…

New Guidelines for Care of Children With Cerebral Palsy
 New Guidelines for Care of Children With Cerebral Palsy 1024 683 Abbie Miller
By: Abbie Miller
Young child with Cerebral Palsy

Guidelines from the American Academy of Pediatrics and the American Academy for Cerebral Palsy and Developmental Medicine highlight advances in diagnosis, care and outcomes for children and adolescents with cerebral palsy. In late 2022, the American Academy of Pediatrics and the American Academy for Cerebral Palsy and Developmental Medicine released new guidelines to help primary…

Exploring the Complex Immune Microenvironment of Rasmussen Encephalitis
 Exploring the Complex Immune Microenvironment of Rasmussen Encephalitis 1024 587 Lauren Dembeck
By: Lauren Dembeck

Researchers used a multi-omics approach to gain a deeper understanding of aberrant cellular signaling leading to progression of a rare childhood neurological disease.   Rasmussen encephalitis is a very rare childhood neurological disease characterized by chronic progressive inflammation of one hemisphere of the brain, leading to loss of function, neurodegeneration, and drug-resistant epilepsy. Researchers have…

Low-Resource Screening Tool Demonstrates Superior Predictive Value for Cerebral Palsy in Infants
 Low-Resource Screening Tool Demonstrates Superior Predictive Value for Cerebral Palsy in Infants 1024 683 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

The use of lower-resource screening tools for cerebral palsy in infants can help increase early detection of the condition and promote access to early interventions.   A recent study reported that the Hammersmith Infant Neurological Examination (HINE), when compared with the Test of Infant Motor Performance (TIMP), had the better predictive value for detecting cerebral…

Using Hormone Therapy First Drives Earlier Remission From Infantile Epileptic Spasms Syndrome
 Using Hormone Therapy First Drives Earlier Remission From Infantile Epileptic Spasms Syndrome 1024 575 Natalie Wilson
By: Natalie Wilson

Recent research conducted at Nationwide Children’s Hospital demonstrated standardizing hormone therapy as the first treatment for infantile spasms improved rates of early remission. Infantile epileptic spasms syndrome (IESS) is an uncommon epilepsy syndrome characterized by seizures called infantile spasms that begin between 1 and 24 months of age (with a peak age of onset at…

Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions
 Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions 1024 575 Lauren Dembeck
By: Lauren Dembeck

The findings contribute to a scant body of literature on the changes in functional connection within the brain and neurocognitive outcomes in survivors of pediatric brain tumors. Pediatric brain tumors are the most common solid tumor diagnosis among children in the United States. With advances in treatment, the 5-year survival rate among these children and…

Quality of Life in Pediatric Patients With Non-ambulatory Cerebral Palsy and Epilepsy
 Quality of Life in Pediatric Patients With Non-ambulatory Cerebral Palsy and Epilepsy 600 400 Mary Bates, PhD
By: Mary Bates, PhD

Study indicates that quality of life in young people with non-ambulatory cerebral palsy decreases with the use of more antiseizure medications. In a new study, researchers from Nationwide Children’s Hospital assessed the relationship between epilepsy-specific characteristics and proxy-reported health-related quality of life in children and young people with non-ambulatory cerebral palsy. The findings indicate that…

Featured Researcher — Allison Bradbury, MS, PhD
 Featured Researcher — Allison Bradbury, MS, PhD 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Allison Bradbury, MS, PhD, is an accomplished principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital, but her introduction to medical research and the field of rare neurologic diseases wasn’t at school — it was in her great uncle’s garage, when his work as a veterinary pathologist led him to identify an inherited…

Featured Researcher — Tracy Bedrosian, PhD
 Featured Researcher — Tracy Bedrosian, PhD 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Tracy Bedrosian, PhD, is a principal investigator in the Steve and Cindy Rasmussen Institute for Genomic Medicine and a specialist in brain mosaicism and neurodevelopment working to uncover the impact that localized mutations in brain cells have on epilepsy symptoms, autism and other neurodevelopmental diseases. She collaborates with neurologists and other experts in the Steve…

Epilepsy Surgery Underutilized in Young Patients With Drug-Resistant Epilepsy
 Epilepsy Surgery Underutilized in Young Patients With Drug-Resistant Epilepsy 1024 575 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Color close up image of young baby girl looking at hanging toys

Research suggests that early surgical intervention can boost developmental outcomes and even cure young children with intractable epilepsy, but low referral rates and other barriers result in limited access for some. Early surgery for drug-resistant epilepsy (DRE) can improve quality of life by reducing or even eliminating seizures. DRE affects about one in every three…

4-Year Study Finds No Correlation Between Head Impacts and Cognitive Outcomes in Youth Tackle Football Players
 4-Year Study Finds No Correlation Between Head Impacts and Cognitive Outcomes in Youth Tackle Football Players 1024 683 Lauren Dembeck
By: Lauren Dembeck

In the longest study of its kind, researchers studied individual players through 4 seasons of youth tackle football and found no correlation between the number or severity of head impacts and cognitive and behavioral outcomes.  When children are considering playing contact sports, parents/guardians and medical providers must weigh the risks and benefits. One of the…

Long-Term Follow-up Study Shows Cannabidiol is Safe and Effective for Lennox-Gastaut Syndrome
 Long-Term Follow-up Study Shows Cannabidiol is Safe and Effective for Lennox-Gastaut Syndrome 695 473 Mary Bates, PhD
By: Mary Bates, PhD
Black and white environmental portrait of Dr. Anup Patel standing in the hospital in his lab coat with his hand in his pocket

Long-term treatment with highly purified plant-based CBD shows continued seizure reduction and no new side effects. The results of a long-term open-label extension trial of plant-derived, highly purified cannabidiol (CBD) show the treatment is effective and safe long term for patients with Lennox-Gastaut syndrome. The treatment had a similar safety profile as in the original…