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2021 VCP Scientific Conference

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Proudly supported by


Tsui-Fen Chou, PhD

Research Professor in Biology at California Institute of Technology

Virginia Kimonis, MD, MRCP

Professor, Division of Genetics and Genomic Medicine, Department of Pediatrics at UC Irvine

Nathan Peck

VCP disease patient and Cure VCP Disease leader

Sujata Patel

Cure VCP Disease Board Member

Hemmo Meyer, PhD

Professor, Department Molekularbiologie, Universität Duisburg-Essen

Alyssa Johnson, PhD

Assistant Professor, Louisiana State University

Peter Shen, PhD

Assistant Professor of Biochemistry, University of Utah

George Tolomiczenki, PhD, MPH, MBA

Executive Director, Merkin Institute for Translational Research at Caltech

Alison Skrinar, PhD

Vice President Clinical Outcomes Research and Evaluation (CORE), Ultragenyx

Barbara Wold, PhD

Director, Merkin Institute for Translational Research at Caltech

Fabio Coppedè, PhD

Associate Professor of Medical Genetics at Università di Pisa

Nicholas Seyfried, PhD

Associate Professor, Emory University School of Medicine

Donna Huryn, PhD, ACSF

Professor, University of Pittsburgh School of Pharmacy

Gerald Pfeffer, MD, CM, FRCPC, PhD

Assistant Professor Clinical Neurosciences, University of Calgary

Jordi Diaz-Manera, MD, PhD

Professor of Neuromuscular Disorders, Newcastle University

Michelle Arkin, PhD

Department Chair and Professor, Department of Pharmaceutical Chemistry, UCSF

Lindsay Alfano, PT, DPT, PCS

Principal Investigator - The Abigail Wexner Research Institute at Nationwide Children’s Hospital

Eddie Lee, MD, PhD

Assistant Professor, Pathology & Laboratory Medicine, UPenn Perelman School of Medicine

Manisha Korb, MD

Assistant Professor — Department of Neurology, UCI School of Medicine

Nelson Pace, PhD, SM

Director of Epidemiology at AllStripes

Tahseen Mozaffar, MD

Professor — Department of Neurology, UCI School of Medicine

Conrad (Chris) Weihl, MD, PhD

Professor of Neurology at Washington University School of Medicine in St. Louis

Jerry Mendell, MD

Professor of Pediatrics and Neurology at Nationwide Children’s Hospital

Meredith James, PT

Research Physiotherapist, John Walton Muscular Dystrophy Research Centre

Kalina Paunovska, PhD

Senior Research Scientist at Dahlman Lab, Georgia Institute of Technology

Thomas Blanchard, PhD, JD

Director of the University of Maryland Brain and Tissue Bank (UMBTB)

Bhaskar Roy, MBBS, MBioMedSci, MHS

Assistant Professor, Yale School of Medicine

Nupur Ghoshal, MD, PhD

Associate Professor, Neurology at at Washington University School of Medicine in St. Louis

The event is over

Hosted by

Cure VCP Disease, Inc.

We are a patient advocacy organization driving efforts to find a cure for diseases caused by a mutation of the VCP gene including IBMPFD and ALS.


Cure VCP Disease, Inc.

Conference Sponsor


Conference Sponsor

Everylife Foundation for Rare Diseases

Conference Sponsor

Coordination of Rare Diseases at Sanford (CoRDS)

Industry Partner


Industry Partner

Po-Lin Chiu, PhD

Structural and functional analyses of disease-linked p97 ATPase mutant

Stephanie Moon, PhD

Coupling of translation quality control and mRNA targeting to stress granules

Alyaa Shmara, MBChB

High Prevalence of Frontotemporal Dementia in Females in Five Hispanic Families with R159H VCP Multisystem Proteinopathy

Alyaa Shmara, MBChB

A Clinicopathologic Study of Malignancy in VCP-Associated Multisystem Proteinopathy

Cheng Cheng, PhD

VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy

Rod Carlo Columbres

Bone density studies using DEXA in patients with VCP myopathy and/or Paget disease of bone

Rod Carlo Columbres

Analysis of quantitative MRI features and functional assessment in IBMPFD patients

Rod Carlo Columbres

Semi-quantitative MRI and functional analysis of thigh muscles in IBMPFD patients

Jacob Klickstein

Modeling p97 ALS using isogenic IPSC-derived motor neurons

Feng Wang, PhD

p97/VCP ATPase inhibitors can prevent p97 mutation-linked motor neuron degeneration

Veronica Ferrari

Insight in VCP mutants pathological mechanisms: disruption of lysosomal stability, multilamellar bodies formation and autophagy activation

Jiang Zhu

Loss of Valosin-Containing Protein (VCP) activity enhances proteopathic seeding

Eiman Abdoalsadig

Utilization of CoRDS Registry to Monitor Quality of Life in Patients with VCP Disease

Manisha Korb, MD

Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy

Carly Pontifex

Examining Interactions Between Autophagy and Stress Granules Dysfunction in a Digenic VCP and TIA1 Distal Inclusion Body Myopathy

Rakesh Ganji, PhD

Regulation of ER-mitochondrial Contact Sites by the p97 AAA-ATPase

Natalie Reash, PT, DPT

Establishing clinical trial readiness for valosin containing protein-associated multisystem proteinopathy: baseline demographics from a 1-year prospective study

Mallory Jones

Overview of Valosin Containing Protein from the Perspective of a Rare Disease Advocacy Organization

Ankita Basu

CRISPR/Cas9-engineered Drosophila knock-in models to study VCP diseases

Brittany Ahlstedt

Regulation of ER-protein homeostasis by the p97-UBXN1 complex

Purbasha Nandi

Structural insights into the p97 disease mutant complex with p47 and its functional implications

Eileen Lynch, PhD

Effects of VCP mutations on endolysosomal damage and TDP-43 aggregation in skeletal muscle and neurons

Katelyn Green, PhD

Mechanisms of stress granule dysregulation caused by VCP mutations

Brenna Powers, PT, DPT

Evaluating consistency of subject performance between 2-day visits in subjects with valosin-containing protein multisystem proteinopathy

Megan Iammarino, PT, DPT

Utility and reliability of patient-reported outcome measures for valosin containing protein-associated multisystem proteinopathy: baseline results from a 1-year prospective study

Ryan Mahoney

Benefits of Remote Inspiratory Exercise Training in Familial Myopathy

Marianela Schiava, MD

Clinical classification of variants in the valosin containing protein gene associated with multisystem proteinopathy

Marianela Schiava, MD

Genotype-phenotype correlations in Valosin Containing Protein disease: Results of an International Multicentric study