HUGO MOSER RESEARCH INSTITUTE OVERVIEW

Kennedy Krieger’s scientists and researchers are leaders in the worldwide effort to prevent and treat disorders of the brain, spinal cord, and musculoskeletal system.  Our investigators continue to break new ground with innovative magnetic resonance imaging technology, investigate critical areas, such as the role of genetics in developmental disorders, and develop new treatment models and therapies. Kennedy Krieger is committed to sharing our knowledge that can be adapted and used by other healthcare organizations to benefit patients across the country and around the world.

Professional Training Overview

Kennedy Krieger dedicates substantial resources to increasing the number of qualified specialists in the fields of neurological and developmental disabilities. We also take seriously our responsibility not only to improve care through research, but to share that knowledge with others. That is why more than 400 individuals come here to train in a wide variety of disciplines each year. The goals of the Institute’s clinical training programs are twofold: 1) to train leaders in the field of neurodevelopmental disabilities and 2) to help ensure that there is a sufficient number of qualified professionals to meet the needs of children with special health care needs and their families. By training future generations of researchers and professionals – from undergraduate students to current staff – we are ensuring that children everywhere can receive the best possible care.

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A National Research Resource Center for Functional Brain Imaging Technology

The only children's hospital designated as a NIH National Research Resource for functional brain imaging, Kennedy Krieger is breaking new ground with innovative fMRI technology.
Understanding Behaviors Through Observational Research

Discovering the environmental and biological basis of behavior is crucial to understanding and improving treatment outcomes for a wide variety of developmental disorders.
Motion Analysis Research and Laboratory

Using 3D modeling and other innovative technologies, our researchers are studying new methods to improve walking patterns in those with brain injuries.
IAN Research: Advancing Research, Empowering Advocates, Interpreting Findings

The Interactive Autism Network (IAN) is the nation's largest online autism research effort. IAN Research collects data from families online and matches them to studies nationwide.

HUGO MOSER RESEARCH INSTITUTE OVERVIEW

Kennedy Krieger’s scientists and researchers are leaders in the worldwide effort to prevent and treat disorders of the brain, spinal cord, and musculoskeletal system.  Our investigators continue to break new ground with innovative magnetic resonance imaging technology, investigate critical areas, such as the role of genetics in developmental disorders, and develop new treatment models and therapies. Kennedy Krieger is committed to sharing our knowledge that can be adapted and used by other healthcare organizations to benefit patients across the country and around the world.

Participate in Research

Over the years, Kennedy Krieger’s scientists and researchers have made crucial medical discoveries that have led to innovative treatments and improved patient care for individuals with disorders of the brain, spinal cord and musculoskeletal system. Learn more about current research studies you can participate in by visiting our recruiting research studies page. Learn more.

Current Research Projects

Mechanisms and Rehabilitation of Cerebellar Ataxia
Principal Investigator: Bastian, Amy
Sponsored by the National Institutes of Health -- 2R01HD040289. Cerebellar damage disrupts movement coordination. Actions ranging from walking to reaching become inaccurate and difficult to control,...
Human Locomotor Plasticity in Health and Disease
Principal Investigator: Bastian, Amy
Sponsored by the National Institutes of Health -- 5R01HD048741-06. Walking patterns can adapt on short timescales (minutes to hours) to temporary conditions, such as icy sidewalks. They can also...
The Development of a Reading Intervention for Deaf and Hard-of-Hearing Students
Principal Investigator: Beetar, John
The specific aim of this project is to complete the development of a computer software program of reading instruction, Sign 'n' Read, for deaf and hard-of-hearing students. Presently in development...
Kennedy Krieger Family Center - Integrated Trauma Approaches
Principal Investigator: Belcher, Harolyn
The goals of the Kennedy Krieger Family Center Trauma Intervention Program (TIP) are to: Evaluate the feasibility and effectiveness of evidence-based treatment protocols, endorsed by the National...
Maternal Child Health Careers/Research Initiatives -- Undergraduate Program
Principal Investigator: Belcher, Harolyn
Sponsored by DHHS/CDC -- 1U50MN000025. Healthy People 2020 highlights the importance of eliminating health disparities and increasing the quality and years of healthy life free of preventable disease...
A 12 Week Double Blind Placebo Clinical Trial to Evaluate the Safety and Efficacy of Rivastigimine in Children with Down Syndrome
Principal Investigator: Capone, George
Individuals with Down syndrome (DS) exhibit a notable cholinergic deficiency by the 4th decade of life. It is unclear how early in life presynaptic cholinergic deterioration begins or how it evolves...
Intellectual and Developmental Disabilities Research Centers 2008
Principal Investigator: Cataldo, Michael
This application for continued support of Years 21 through 25 of the Mental Retardation and Developmental Disabilities Research Center (MRDDRC) at the Kennedy Krieger Institute and Johns Hopkins...
Developing a Means of Comparing Expenditures at Three Models of Nonpublic Special Education Services at a Private Special Education School in Baltimore, Maryland
Principal Investigator: Church, Robin
Currently, there is no uniform means of determining the true cost of nonpublic special education in Maryland. Today, tuition is used as “the cost” of providing these programs. The proposed project...
Anticonvulsants, Ischemic Seizures and Regeneration in the Immature Brain
Principal Investigator: Comi, Anne
Neonatal stroke affects one in 4000 term births and frequently results in cognitive impairments. Neonatal strokes present with seizures, and anticonvulsants (usually phenobarbital) are administered...
Behavioral Economics in MR/DD and Behavior Disorders (2)
Principal Investigator: DeLeon, Iser
Assessments of severe behavior disorders of children with intellectual and developmental disabilities (IDD) have revealed that behavior problems are often maintained by their functional outcomes (or...
Translational Analyses of Chronic Aberrant Behavior Across the Lifespan
Principal Investigator: DeLeon, Iser
The goal of the proposed line of research is to gain a better understanding of the processes that evoke and maintain severe problem behavior (i.e., chronic aberrant behavior) in persons with...
EEG-Based Assessment of Functional Connectivity in Autism
Principal Investigator: Ewen, Joshua
Sponsored by the National Institutes of Health -- 1K23NS073626-01. There are substantial data to suggest that altered cerebral connectivity is a key aspect of the neurobiology of autism. Nevertheless...
Study of White Matter Development in a Rodent Model of Perinatal Brain Injury
Principal Investigator: Fatemi, S. Ali
This is a Mentored Clinical Scientist Research Career Development Award allowing Dr. Fatemi to develop a career as an independent investigator in the field of translational neuroscience research,...
Comprehensive Long-Term Evaluation of the Gunn Rat Kernicterus Model
Principal Investigator: Fatemi, Seyed A.
The proposed project is the initial phase of a long-term endeavor to develop therapies that will help patients with dyskinetic cerebral palsy related to mitochondrial dysfunction and a Kernicterus-...
Mentored Clinical Scientist Research Career Development Award
Principal Investigator: Fatemi, Seyed A.
The proposed training will prepare a young pediatric neurology faculty member for a career as an independent investigator in the field of translational neuroscience research, focusing on perinatal...
An adaptive, phase IIB/III, double-blind, randomized, placebo-controlled, multi-center study of the safety and efficacy of NaBen® (sodium benzoate), a d-amino acid oxidase inhibitor, as an add-on treatment for schizophrenia in adolescents
Principal Investigator: Findling, Robert
Phase IIb Objectives: The primary objective of the Phase IIb portion of this study is to evaluate NaBen® (sodium benzoate) (1000 mg/day) safety and effectiveness and to determine the final sample...
Validation Study of the Aggression Diary
Principal Investigator: Findling, Robert
The primary objective of this study is to conduct psychometric tests on the electronic observer- reported (eObsRO) Aggression Diary to evaluate whether the diary is a valid and reliable measure for...
Activin Receptor-Based Therapies for Musculoskeletal Disease
Principal Investigator: Germain-Lee, Emily
Sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Disease -- 1R01AR062074-04 The overall goal of this project is to investigate the role of the myostatin/activin signaling...
Phase 2 of Growth Hormone for Treatment of Albright Hereditary Osteodystrophy
Principal Investigator: Germain-Lee, Emily
Sponsored by the Food and Drug Administration -- 5R01FD003409-02. Albright hereditary osteodystrophy (AHO) is a rare genetic disorder caused by heterozygous inactivating mutations in the gene...
Osteogenesis Imperfecta Clinic
Principal Investigator: Germain-Lee, Emily
Sponsored by the Harry Headley Charitable & Research Foundation. Funding for an independent clinician/researcher to work with Dr. Jay Shapiro in the OI Clinic.
Retrospective Chart Review of Growth in Osteogenesis Imperfecta
Principal Investigator: Germain-Lee, Emily
The purpose of this research is to assess the growth parameters(height and weight) in osteogenesis imperfecta during infancy, childhood, and adolescence with the ultimate goal of determining whether...
Reward System in Autism
Principal Investigator: Goldberg, Melissa
It is a core feature of autism that social interactions appear to be far less rewarding for autistic children compared to typically developing children. Based on this observation, one would predict...
An evaluation of differential reinforcement on appropriate toy skills
Principal Investigator: Gonzales, Melissa
The purpose of the current study will be to increase the play skills for six children with developmental disabilities, who display limited interest in toys in a free operant setting. It is...
Delineating subtypes of self-injurious behavior maintained by automatic reinforcement
Principal Investigator: Hagopian, Louis
Self-injurious behavior (SIB) is one of the most serious problems experienced by individuals with intellectual disabilities. The standard of care for the assessment of SIB involves conducting a "...
Quantitative mapping of the basal ganglia and related structures in children with dyskinetic cerebral palsy
Principal Investigator: Hoon, Alexander
Cerebral palsy (CP) refers to a group of motor-impairment syndromes secondary to genetic and acquired disorders of early brain development.  When the underlying disorder affects the basal...
Investigation of Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
Principal Investigator: Tierney, Elaine
This is not a new study, but rather a re-submission of a previously approved study for which IRB approval lapsed. The study is closed to enrollment, but will remain open so that we can 1) continue...
Data Analyses for the Establishment of The Nail Patella Syndrome (NPS) Behavioral Phenotype
Principal Investigator: Tierney, Elaine
Subjects will not be seen at KKI as part of this study. This study will analyze data collected as part of the previously approved and now closed RPN 00-06-03-05 (entitled, The Behavioral Phenotype of...
Guanfacine in the treatment of hyperactivity and implusiveness in children and adolescents with pervasive development disorders
Principal Investigator: Tierney, Elaine
This application is a request for transfer of this protocol from paper IRB to e-irb submission review: Previous Application Number (paper based submission): 01-04-06-04 Protocol Title: Guanfacine in...
Methylphenidate in the Treatment of Hyperactivity and Implusiveness in Children and Adolescents with Pervasive Developmental Disorders
Principal Investigator: Tierney, Elaine
This application is a request for transfer of this protocol from paper IRB to e-irb submission review: This trial has ended. No children will be enrolled or participate in a clinical trial. We...
The Frequency of Smith-Lemli-Opitz Syndrome (SLOS) in the Population with Autism
Principal Investigator: Tierney, Elaine
The investigators seek to examine specimens gathered by the Autism Genetic Resource Exchange (AGRE) from individuals with autism spectrum disorders (ASD) and their family members (when available),...
The Behavioral Phenotype of Smith-Lemli-Opitz-Syndrome and Other Sterol Disorders
Principal Investigator: Tierney, Elaine
The primary goals of this project are to 1) determine the cognitive, social, and communication abilities and to define the behavioral phenotype of a spectrum of genetically and biochemically well-...
A Study of Risperidone in Children, Adolescents and Young Adults with Autistic Disorder
Principal Investigator: Tierney, Elaine
This study seeks to reactivate the following protocols under one new RPN during the post-study analyses phase of these studies: 04-06-03, 99-04-06-02, 99-05-14-01, and 01-04-16-04. Due in part to...
Cholesterol in ASD II
Principal Investigator: Tierney, Elaine
The purpose of this application is to allow for continued data cleaning of the identifiable data collected under terminated study NA_00020928 (PI Tierney; IRB2), including receipt of data from the...
Brief intensive therapy for children with cerebral palsy: outcomes of a clinical model
Principal Investigator: Trovato, Melissa
Does the function of children with cerebral palsy improve after a brief intensive course of therapy services involving daily physical and occupational therapy. Children's functional skills will be...
Glycogen Quantification using MRI
Principal Investigator: Van Zijl, Peter
The goal of this research is to develop and validate a novel MRI method for imaging glycogen contents of tissue. Glycogen is the main energy storage molecule of the body. Its non-invasive detection...
Quantitation of the BOLD Effect in Functional MRI
Principal Investigator: Van Zijl, Peter
The ultimate goal of this project is to gain a complete quantitative understanding of the physiological, physical, and spatial aspects of the blood-oxygen-level-dependent (BOLD) functional MRI (fMRI...
Glucose as contrast agent for MRI
Principal Investigator: Van Zijl, Peter
MRI assessment of tissue perfusion is currently mainly done using GdDTPA contrast enhancement. This contrast agent is FDA approved and used routinely throughout the country in clinical MRI scans of...
Investigation of Emotional Attention and Memory in Children with Anxiety Disorders
Principal Investigator: Vasa, Roma
This study examines emotional attention and memory in children with anxiety disorders. The data from this study will be compared to data on similar cognitive processes in control children (being...
Effects of manipulating attention on the neural response to threat stimuli in children with and without anxiety disorders
Principal Investigator: Vasa, Roma
This study examines whether anxious children exhibit different patterns of brain activation when attending to threatening stimuli compared to healthy control children. 
Investigation of emotional attention and memory in healthy children
Principal Investigator: Vasa, Roma
This study is collecting data on emotional attention and memory in psychiatrically healthy children. The data from this study will be compared to data on similar cognitive processes in anxious...
Investigating Patterns of Emergency Department Use in Individuals with Autism Spectrum Disorder
Principal Investigator: Vasa, Roma
This protocol requires permission to utilize a de-identified research dataset: the Nationwide Emergency Department Sample (NEDS). Access to these data will advance the understanding of autism...
Psychiatric, Behavior, and Cognitive Profiles in Children with Autism Spectrum Disorder
Principal Investigator: Vasa, Roma
This protocol requests permission to have access to a de-identified research dataset that was collected by the Simons Foundation, a private foundation in New York that focuses on advancing research...
Psychiatric Crisis among Youth and Transition Age Adults with Autism Spectrum Disorder
Principal Investigator: Vasa, Roma
The current proposal is a request to assemble a focus group comprised of multidisciplinary clinicians (e.g., psychiatrists, psychologists, social workers) both within and outside the Kennedy Krieger...
Gene Expression and Genetic Mental Retardation (MR)
Principal Investigator: Vaurio, Rebecca
This project investigates the patterns of gene expression, and associated molecular events, in a sample of boys with genetic mental retardation (MR). We intend to identify profiles of gene expression...
Methylation of Genes in Fetal Alcohol Spectrum Disorders
Principal Investigator: Vaurio, Rebecca
The goals of this project are as follows: 1. Examine methylation profiles in children diagnosed with FASD compared to children with other psychiatric disorders to evaluate whether there are...
Facioscapulohumeral disease biomaterial for Wellstone Core
Principal Investigator: Wagner, Kathryn
An NIH funded, Senatory Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC) has recently been established entitled "Biomarkers for therapy of FSHD (facioscapulohumeral muscular...
Duchenne Muscular Dystrophy: Double-Blind Randomized Trial to Find The Optimum Steroid Regimen (FOR-DMD)
Principal Investigator: Wagner, Kathryn
This Phase III multi-center, double-blind, parallel group, 36 - 60 month study funded by the NIH will compare three corticosteroid regimens in wide use in DMD: 1) daily prednisone (0.75 mg/kg/day); 2...
Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
Principal Investigator: Wagner, Kathryn
This study, supported by the FSH Society, will utilize magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to develop non-invasive, quantitative imaging biomarkers for...
CHAR0312: Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
Principal Investigator: Wagner, Kathryn
The purpose of this research is to collect blood and skin samples from Duchenne Muscular Dystrophy (DMD) patients ages 4 years and above with specific genetic mutations within the dystrophin gene...
Collection of Data from Muscle Strength Testing
Principal Investigator: Wagner, Kathryn
The purpose of this protocol is to collect clinical data on muscle strength and function for informed clinical trial designs. The data will be collected from patients with muscular dystrophy during...
A Phase 1b Open-Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HT-100 in Patients with Duchenne Muscular Dystrophy (HALO-DMD-01)
Principal Investigator: Wagner, Kathryn
This is an open label, single ascending dose (SAD) and multiple ascending dose (MAD) study to evaluate the safety, tolerability, and pharmacokinetics of HT-100, a delayed-release, orally delivered...
An open-label extension study of HT-100 in patient with Duchenne Muscular Dystrophy who have completed Protocol HALO-DMD-101
Principal Investigator: Wagner, Kathryn
This is an open-label, multiple dose extension study to evaluate the safety, tolerability, PD signals, and population PK of HT-100, a delayed-release, orally-delivered agent in participants with DMD...
A Continued Access protocol for eligible US subjects with Duchenne Muscular Dystrophy who previously participated in an approved drisapersen study
Principal Investigator: Wagner, Kathryn
This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with DMD having dystrophin mutations correctable by drisapersen induced DMD Exon 51...
A Three Dimensional Environment for Skeletal Muscle Stem Cell Transplantation
Principal Investigator: Wagner, Kathryn
The purpose of this collaborative project is to develop and test and injectable scaffold embedded with muscle stem cells and a Pfizer drug to facilitate muscle regeneration. Cell based therapies...
An exploratory study to assess two doses of GSK2402968 in the Treatment of Ambulant boys with Duchenne Muscular Dystrophy
Principal Investigator: Wagner, Kathryn
This is a multi-center phase II, double blind, exploratory parallel-group, placebo-controlled clinical study in ambulant subjects with Duchenne muscular dystrophy resulting from a mutation that can...
Bone health in Facioscapulohumeral muscular dystrophy: A cross-sectional study
Principal Investigator: Wagner, Kathryn
This is a cross-sectional single visit study to determine bone health in individuals with FSHD. The measures of bone health, including bone mineral density and lean body mass via DEXA scan and blood...
Assessment of Functional Outcomes of an Interdisciplinary Pediatric Pain Rehabilitation Program
Principal Investigator: Ward, Cynthia
Pediatric chronic pain can result in functional impairments that may negatively impact ones ability to perform daily activities. Pediatric chronic pain patients may benefit from mutlidisciplinary...
Acupressure and Acupuncture as an Intervention with Children with Autism II
Principal Investigator: Warren, Lana
The purpose of this feasibility study is to see if children with autism spectrum disorder (ASD) will tolerate an acupressure and acupuncture intervention twice weekly over a 3 month period of time...
Role of acyl-CoA synthetases in lymphangioleiomyomatosis (LAM)
Principal Investigator: Watkins, Paul
LAM is characterized by proliferation of smooth muscle cells in the lung. SLC27A3 is an acyl-CoA synthetase whose activity seems to be increased in rapidly proliferating cells. The fundamental...
Evaluation of Child, Parent and Physician Characteristics Associated with Complementary and Alternative Medicine Use in Children with Autism Spectrum Disorders
Principal Investigator: Wilms-Floet, Anna Maria
Our research plan is to identify child-, parent- and clinician characteristics that are associated with CAM use, using de-identified, retrospective data from the Autism Treatment Network Registry (...
Differences in Behavioral and Adaptive Profiles among Children Diagnosed with Autism Spectrum Disorders, Attention-Deficit/Hyperactivity Disorder, or Anxiety Disorder
Principal Investigator: Wodka, Erica
This study is a retrospective review of assessment data from routine psychological or neuropsychological evaluations. The focus of this study is to examine behavioral and adaptive profiles of...
Investigating the neurobehavioral basis of sensory behavior in ASD
Principal Investigator: Wodka, Ericka
Autism Spectrum Disorder (ASD) is a group of developmental disorders which affects approximately 1% of children. One of the prominent associated symptoms of ASD is abnormal sensory behaviors. While...
Ecological validity of neuropsychological testing and screening
Principal Investigator: Zabel, Andrew
Neuropsychological assessment and screening is a useful means of identifying cognitive deficits in children and adolescents. Expansion of availability of instruments has now permitted an examination...
Evaluation of data obtained from a Phase III trial to evaluate Lorenzo's oil in adults with X-linked adrenoleukodystrophy
Principal Investigator: Zackowski, Kathleen
This study will evaluate data collected in a placebo-controlled study of Lorenzo's oil in adult men and women with X-linked adrenoleukodystrophy. This study was originally run from 2005-2008 under Dr...
Exercise study of function and pathology for women with X-ALD
Principal Investigator: Zackowski, Kathleen
This study will comprehensively evaluate the corticospinal tract and the dorsal column-medial lemniscal tracts across the brain and cervical spinal cord with the purpose of understanding mechanisms...
Evaluating intervention responsiveness in people with multiple sclerosis
Principal Investigator: Zackowski, Kathleen
Multiple sclerosis (MS) is an inflammatory disease of the central nervous system (CNS). It is characterized by a progressive functional decline although the clinical course of the disease varies...
Effects of training on caregiver implementation of behavior-analytic skills
Principal Investigator: Zarcone, Jennifer
This small N study will examine additional effects of our standard of care for caregiver training on the implementation of specific skills with children with severe problem behavior. These skills...

Faculty & Research Scientists