2021 VCP Scientific Conference
Proudly supported by
Speakers






Booths

Cure VCP Disease, Inc.
Conference Sponsor

AllStripes
Conference Sponsor

Everylife Foundation for Rare Diseases
Conference Sponsor

Coordination of Rare Diseases at Sanford (CoRDS)
Industry Partner

Healx
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