Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study
The study has determined that the NSAD and other assessments are valid and reliable measures for quantifying the level of disease impairment in individuals with LGMDR1.
Authors
- Nikia Stinson
- Shelley Mockler
- Amanda Clause (ORCID: https://orcid.org/0000-0002-2078-7280)
- Nicholas E. Johnson (ORCID: https://orcid.org/0000-0002-3917-4257)
- Robert Will
- Molly M. Stark
- Talia R. Strahler
- M. Iammarino (ORCID: https://orcid.org/0000-0003-2800-3855)
- Conrad C. Weihl (ORCID: https://orcid.org/0000-0002-3816-6124)
- Lindsay N. Alfano (ORCID: https://orcid.org/0000-0002-2263-7569)
- M. James (ORCID: https://orcid.org/0000-0001-8078-7744)
- Aileen Jones (ORCID: https://orcid.org/0009-0003-2882-3025)
- Natalie F. Reash (ORCID: https://orcid.org/0000-0002-0573-9504)
- Stacy Dixon
- Renee Augsburger
- Katherine D. Mathews (ORCID: https://orcid.org/0000-0003-4160-0435)
- Peter B. Kang (ORCID: https://orcid.org/0000-0002-4270-7325)
- Jeffrey Statland (ORCID: https://orcid.org/0000-0003-0790-5315)
- Katie M. Laubscher
- Lindsay Pietruszewski (ORCID: https://orcid.org/0000-0003-0691-5378)
- Sandhya Sasidharan
- Doris G. Leung (ORCID: https://orcid.org/0000-0002-2558-7798)
- Melissa Currence
- Matthew Wicklund (ORCID: https://orcid.org/0000-0003-1198-9363)
- Tahseen Mozaffar (ORCID: https://orcid.org/0000-0002-1230-0188)
- Stephanie Hunn (ORCID: https://orcid.org/0009-0006-0064-9178)
- Amanda Butler (ORCID: https://orcid.org/0009-0009-1809-3341)
- Linda Lowes (ORCID: https://orcid.org/0000-0003-4206-0557)
- R. Horton
Institutions
- Kennedy Krieger Institute (US)
- Nationwide Children's Hospital (US)
- Virginia Commonwealth University (US)
- University of California, Irvine (US)
- Washington University in St. Louis (US)
- Newcastle upon Tyne Hospitals NHS Foundation Trust (GB)
- University of Kansas Medical Center (US)
- University of Minnesota Medical Center (US)
- University of Iowa Health Care (US)
- The Ohio State University (US)
- Newcastle University (GB)
- University of Colorado Denver (US)
Publication Details
- Journal
- Annals of Clinical and Translational Neurology
- Published
- 2025-04-16
- DOI
- https://doi.org/10.1002/acn3.70049
- Citations
- 1
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81
Funders
- Jain Foundation
- Pfizer
- Sanofi
- Muscular Dystrophy Association
- Biogen
- Alexion Pharmaceuticals
- CSL Behring
- Cumberland Pharmaceuticals
- Virginia Commonwealth University
- Seattle Children's Research Institute
- Vertex Pharmaceuticals
- Children’s Hospital of Wisconsin Research Institute
- PTC Therapeutics
- REGENXBIO
- Sarepta Therapeutics
- Amicus Therapeutics
- Harmony Biosciences
- Argenx
- Newcastle University
- Italfarmaco
- National Institutes of Health
- Sanofi Genzyme