Funded Research Projects

Advancing Care With Exon 17 Skipping Therapy for Duchenne Muscular Dystrophy

In Progress

Recipient: Liubov Gushchina, PhD

Nationwide Children's Hospital

Role of Macrophage MCT1 in Diabetic Peripheral Neuropathy

In Progress

Recipient: Brett Morrison, MD, PhD

Johns Hopkins University

Acquired Mitochondrial Dysfunction in Diabetic Neuropathy

In Progress

Recipient: Christopher Cashman, MD, PhD

Mass General Brigham

Evaluation of Aberrant Autophagy and Myogenesis in GNE Myopathy

In Progress

Recipient: Rebecca Schmitt, PhD

Indiana University School of Medicine

Analysis of a Novel Primary Periodic Paralysis SCN4A Mutation With Pain as a Major Phenotype

In Progress

Recipient: Tyler Nelson, PhD

University of Florida

Causal Genetic Variation and Transcriptomic Signatures of the Peripheral Immune System in the Central Nervous System Pathology of ALS That Are Conserved or Divergent Among ALS Patients and the Animal Models

In Progress

Recipient: Mai Yamakawa, MD

UCLA

Genetically Decoding Human Afferent and Efferent Autonomic Ganglia

In Progress

Recipient: Erika Williams, MD, PhD

Massachusetts General Brigham

CRISPR/Cas9 Correction of a Common Duchenne Muscular Dystrophy (DMD) Deletion

In Progress

Recipient: Stefan Nicolau, MD

Abigail Wexner Research Institute at Nationwide Children's Hospital

The Role of Mitochondria in Inclusion Body Myositis

Recipient: Elie Naddaf, MD

Mayo Clinic, Rochester, MN

Activity-Based Treatments to Accelerate Peripheral Axon Regeneration

Recipient: Colin Franz, MD, PhD

Shirley Ryan AbilityLab/Northwestern University, Chicago, IL

Neuromuscular Ultrasound and Electric Impedance Myography in CMT type 1A: A Prospective Longitudinal Study

Recipient: Yohei Harada, MD

Duke University, Durham, NC

Determining the Pathogenicity of Autoantibodies in Idiopathic Small Fiber Neuropathy

Recipient: Alexander Chamessian, MD, PhD

Washington University School of Medicine, St. Louis, MO

Neurological Application of Neurotoxins

Recipient: R. James Cotton, MD, PhD

Northwestern University and Shirley Ryan AbilityLab, Chicago, IL

Prevalence and Spectrum of Adult Acute Flaccid Paralysis in Zambia

Recipient: Michelle Kvalsund, MD

Michigan State University, East Lansing, MI

Chronic Inflammatory Demyelinating Polyneuropathy Research Fellowship

Recipient: Karissa Gable, MD

Duke University Medical Center, Durham, North Carolina

Androgen Receptor Isoforms: From SBMA Pathogenesis to Therapeutic Targets

Recipient: Carlo Rinaldi, MD, PhD

Associate Professor Department of Physiology, Anatomy and Genetics Division of Medical Sciences, University of Oxford, United Kingdom

Positron Emission Tomography (PET) Imaging for Characterizing Neuroinflammation of Spinal Cord in Amyotrophic Lateral Sclerosis (ALS)

Recipient: Suma Babu, MBBS, MPH

Instructor in Neurology, Harvard Medical School, Massachusetts General Hospital

The Value of High Quality Electrodiagnostic Study in Work-Associated Carpal Tunnel Syndrome

Recipient: Teryl K. Nuckols, MD

RAND Corporation

Validation of the Triple Timed-Up-and-Go Test as a measure of disease severity in Lambert Eaton Myasthenia patients

Recipient: Shruti Raja, MD

Duke University Medical Center, Durham, North Carolina

Neurological Application of Neurotoxins

Recipient: Lisa Williams, MD

University of California, Davis Medical Center, Davis, California

Motor System Connectivity Influences in Amyotrophic Lateral Sclerosis

Recipient: Christi L Kolarcik, PhD

University of Pittsburgh, Pittsburgh, PA

Glucocorticolds in Fiber Repair and Regeneration of Dystrophic Muscles

Recipient: Mattia Quattrocelli, PhD

Center for Genetic Medicine, Northwestern University, Chicago, IL

Cognition in Duchenne Muscular Dystrophy

Recipient: Mathula Thangarajh, MD

Virginia Commonwealth University, Richmond, VA

Improving the Diagnosis of Neuromuscular Diseases

Recipient: Monkol Lek, PhD

Massachusetts General Hospital, Boston, MA

LncRNA as Therapeutic Target for SMA

Recipient: Constantin d'Ydewalle, PhD

Johns Hopkins School of Medicine, Baltimore, MD