Coalition to Cure Calpain 3 (C3) is pleased to announce that Stephanie Hunn DPT was awarded a Travel Grant for the presentation of her research at the Annual Congress of the World Muscle Society in Charleston, South Carolina, from October 3-7, 2023. The goal of the C3 Travel Grant program is to foster early career investigators who are currently conducting research in the area of LGMD2A/R1 or Calpain 3. The grants support travel expenses to attend relevant conferences to present the investigator’s ongoing work.
Dr. Hunn is a Clinical Research Specialist in Dr. Conrad Weihl’s research group at the Washington University School of Medicine. Her poster showed longitudinal data from the LGMD2A/R1 cohort within the GRASP-LGMD Consortium* natural history study. This multi-site study aims to understand disease progression by identifying and measuring clinical outcome assessments that can be used in future interventional clinical trials. With potential treatment trials on the horizon, it is imperative that we understand how to detect changes in disease symptoms over time. The work is being funded by C3, the Muscular Dystrophy Association, and the National Institutes of Health.
Study participants visited the clinic for assessments at baseline, 6-months, and 12-months. Dr. Hunn and colleagues identified the North Star Assessment for Limb-Girdle Type Dystrophies (NSAD) and Performance of Upper Limb (PUL) as clinical outcomes assessments that can detect disease progression over one year. These results represent early data from the first 41 participants to enroll in the study. The target enrollment for the study is 70. Individuals living with LGMD2A/R1 who are able to ambulate independently and are interested in joining can learn more here or by contacting Ruby Langeslay at Ruby.Langeslay@vcuhealth.org
*Additional poster authors:
Andrew R. Findlay1, Lindsay N. Alfano2, Linda P. Lowes2, Mathew Wicklund5, Katherine D. Mathews6, Tahseen Mozaffar7, Doris Leung8, Aileen Jones3, Amanda Butler3, Melissa Hayes4, Sandhya Sasidharan4, Natalie F. Reash2, Megan A. Iammarino2, Katie Laubscher6, Shelley Mockler6, Renee Ausberger7, Megan Holzer8, Nikia Stinson8, Michelle Seiffert1, Jeffrey Statland4, and Nicholas E. Johnson3
1Washington University in St. Louis; 2Nationwide Children’s Hospital; 3Virginia Commonwealth University; 4Kansas University Medical Center; 5University of Colorado—Denver; 6University of Iowa; 7University of California—Irvine; 8Kennedy Krieger Institute