Funded Research Projects

Aging and Dementia in Adults with Down Syndrome
Principal Investigator: Silverman, Wayne
This ongoing NIH supported Program Project will be renewed for another five years, and is programmatically a seamless continuation of my current Exempt protocol (#NA_00002225; "Changes in Functioning...
Drug Discovery for AMN
Principal Investigator: Steinberg, Steven
The overall goal is to accelerate the pace at which new therapies for the most common inherited peroxisomal disorder, adrenomyeloneuropathy (AMN), are discovered and tested in clinical trials through...
Neural Basis of Recovery of Inhibitory Control After Pediatric TBI
Principal Investigator: Suskauer, Stacy
Sponsored by NIH/NICHD - 5 K23 HD061611 Inhibitory control is frequently impaired following pediatric traumatic brain injury (TBI), and deficits in inhibitory control impact a child's function in...
Biomarkers of mild traumatic brain injury in children: predicting outcome and understanding recovery
Principal Investigator: Suskauer, Stacey
Scientific Abstract: Current management of childhood mild TBI (mTBI, also known as concussion) is symptombased. Symptoms may last from hours to years, yet we lack objective biomarkers with which to...
Annual Meeting of the NCRR/NIBIB Principal Investigators
Principal Investigator: Van Zijl, Peter
We propose to continue the annual meetings of the Directors and Assistant Directors of the NCRR and NIBIB P41 Centers. The 2 1/2 day meeting is held annually in late June in Bethesda, Maryland. The...
Resource for Quantitative Functional MRI
Principal Investigator: Van Zijl, Peter
The Resource for Quantitative Functional Magnetic Resonance Imaging is an interdepartmental and interdisciplinary laboratory combining facilities of the F.M. Kirby Research Center for Functional...
Novel Approaches for CEST Labeling, Detection, Quantification, and Translation
Principal Investigator: Van Zijl, Peter
The overall Program Project focuses on the design and detection of bioorganic biodegradable Chemical Exchange Saturation Transfer (CEST) agents, with the ultimate goal of their practical application...
Magnetic Resonance Spectroscopy Imaging as a Biomarker for FSHD
Principal Investigator: Wagner, Kathryn
Sponsored by the American Academy of Neurology -- 2011 AAN Clinical Research Training Fellowship. Recent advances in the study of heritable neuromuscular disorders have led to multiple promising...
A Novel Xenograft Mouse Model of Facioscapulohumeral Muscular Dystrophy
Principal Investigator: Wagner, Kathryn
Despite being one of the most prevalent neuromuscular disorders worldwide, there have been very few clinical trials in Facioscapulohumeral muscular dystrophy (FSHD). Currently there are no clinical...
Biomarkers of Facioscapulohumeral Muscular Dystrophy (FSHD)
Principal Investigator: Wagner, Kathryn
This NIH-funded study is established to identify biomarkers for facioscapulohumeral muscular dystrophy (FSHD). FSHD patients and their unaffected first degree relative will undergo open muscle biopsy...
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...
HALO-DMD-01: A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) of HT-100 in Patients with Duchenne Muscular Dystrophy
Principal Investigator: Wagner, Kathryn
The study, sponsored by HALO Therapeutics, is designed to provide initial safety, tolerability, and PK data of halofuginone hydrobromide, HT-100, in young boys ages 6-20 years with DMD.  HT-100...
HALO-DMD-02: An Open Label Extension Study of HT-100 in Patients with Duchenne Muscular Dystrophy who have Completed Protocol HALO-DMD-01
Principal Investigator: Wagner, Kathryn
This open-label, multiple dose extension study is designed to evaluate the safety, tolerability, pharmacodynamic signals, and population PK of HT-100, a delayed-release, orally-delivered agent in...
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...
Myostatin regulates fate of satellite cells in dystrophic muscle
Principal Investigator: Wagner, Kathryn
The goal of this proposal is to determine whether myostatin, expressed in both skeletal muscle and fibroblasts, regulates the conversion of satellite cells to fibroblasts and whether this conversion...
Partnering to treat an Orphan Disease Duchenne Muscular Dystrophy
Principal Investigator: Wagner, Kathryn
The goal of this program is to develop an investigational drug by Sanofi for a new indication, Duchenne Muscular Dystrophy.  The program includes preclinical toxicology and efficacy studies as...
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...
Biomarkers for Therapy of FSHD
Principal Investigator: Wagner, Kathryn
The goal of this Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center is to define modifying genes of FSHD and to determine, through novel animal models of FSHD, whether these are...
Acyl-CoA Synthetase ACSVL3 in Malignant Glioma: Metabolism and Oncogenic Cellular
Principal Investigator: Watkins, Paul
Malignant glioma accounts for a significant percentage of brain tumors. As these tumors are typically refractory to treatment, there is a need for new and novel therapeutic approaches. Levels of...
Understanding Mechanisms of Lower Extremity Strengthening in Women Heterozygous for X-ALD
Principal Investigator: Zackowski, Kathleen
Sponsored by the ELA Foundation. X-linked adrenoleukodystrophy (X-ALD), a sex-linked progressive neurodegenerative disease, is caused by a defect in the ABCD1 gene. The disease is expressed in...
Predicting Intervention Responsiveness to Improve Rehabilitation in People with Multiple Sclerosis
Principal Investigator: Zackowski, Kathleen
Sponsored by the National Multiple Sclerosis Society -- RG 4668-A-1. Multiple sclerosis (MS) is an inflammatory disease of the central nervous system (CNS) distinguished pathologically by...