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MDCRN
Home
GRASP
Network Information
Active Studies
Past Studies
Biorepository
Site and Principal Investigator List
Newsletters
DMCRN
Network Information
Active Studies
Site and Principal Investigator List
Newsletters
Community Engagement
Limb-Girdle Muscular Dystrophy
Patient Education Day
Patient Education Day Information
Patient Education Day Agenda
Patient Day Videos
Conference
Conference Info
Conference Documentation
Conference Videos
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November 8, 2023

GRASP Protocol Overviews and Updates
Variant Resolution Updates
Novel Genes an Nanopore Sequencing
FKRP Data Presentations
Remote Outcomes update
MRI
Pilot project updates
CDM / ChDM outcomes
Welcome and DMCRN Protocol Review
Re-generating Normative data with QMT and HHD

November 9, 2023

Composite Splicing Biomarker for DM1: Updates and Next Steps
DUX4 minipigs for FSHD
Insights into Spatiotemporal Pathogenesis from Knock-In Mouse Models of Myotonic Dystrophy Type 1
FSHD Composite Outcome Measure
The Continued Promise of Genomic Technologies and Software in Neuromuscular Genetics
Therapeutic Testing in Models of LGMD
Functional Endpoints in Myotonic Dystrophy Type 1
The NSAD in LGMD
MDCRN Educational Day Introduction FSHD
A Micro-Dystrophin Origin Story
Genetic Modifiers of Severity in Huntington Disease
Use of Composite Outcome Measures in Clinical Trials
Development of Outcome Measures in DMD
Genetic Modifiers of Severity in Duchenne Muscular Dystrophy
Under Represented Minority Considerations
Developing Training Programs for the Next Generation
Recruitment in Rare Diseases

November 10, 2023

Opening Remarks
Biorepository Updates
Data Management and Quality Assurance
ReSolve Dyne EU Baseline Data
ReSolve Late Onset Cohort
MOVE Methylation Data
Revised RWS Analysis
MOVE+ Preliminary MRI Analysis
Pediatric Outcomes for FSHD
Closing Remarks
MOVE Baseline Data
MOVE Remote Pilot & Actigraph Data