What is a natural history study? A natural history study is a research study that collects information about the genetics, symptoms, and lived experiences of individuals. These studies are considered observational, because they do not include an interventional drug or
Sarepta Therapeutics recruits for clinical outcomes assessment study
C3 is pleased to share this opportunity on behalf of Sarepta Therapeutics: Sarepta welcomes individuals with LGMD2A/R1 to join a clinical outcomes assessment study. To learn more about the study, visit journeyLGMD.com
New natural history study recruiting individuals with LGMD2A/R1 in France
Coalition to Cure Calpain 3 is pleased to share information regarding a natural history study being conducted in France. Patients must be affiliated with French social security to participate. Additional inclusion criteria is detailed in the flyer below. If you
WATCH: Sarepta presents webinar about new natural history study for individuals living with LGMD2A/R1
Sarepta Therapeutics recently joined C3 to present a community webinar titled “Journey Update for the LGMD2A/R1 Calpainopathy Community.” During the webinar, Sarepta discussed the essential role for natural history studies throughout the drug development process. They then shared the design
C3 hosts Sarepta to present webinar about the JOURNEY natural history study
Click here to register!
Trial readiness study for individuals with LGMD2A/R1
Please contact either Ruby Langeslay or Jennifer Raymond at Virginia Commonwealth University for more information
Casimir Recruiting LGMD2A/R1 Patients for At-Home Study
The Casimir LGVA study is quickly filling! We are specifically seeking individuals with LGMD2A who can walk across a room and stand up from sitting on a couch to participate. Email LGMDS@casimirtrials.com or call (800)542-0948 for more information.
Have you participated in a clinical research study?
Attention Individuals with LGMD2A/R1: Coalition to Cure Calpain 3 wants to hear from you! If you have participated in clinical research, such as a natural history study, an observational study, or a clinical trial, please follow this link to answer
Will YOU help advance LGMD2A/R1 research by participating in the GRASP-LGMD Natural History Study?
Natural history studies collect information about the symptoms and progression of a disease in the absence of an intervention. It is vital to understand natural history so that researchers can plan for clinical trials to determine if an investigational drug
LGMD2A/R1 Patient Enrollment Underway for C3-Sponsored Natural History Study
The GRASP-LGMD consortium is currently recruiting for a natural history study for individuals with mutations in CAPN3 (calpain 3). This study will inform the design of future therapeutic trials and a better understanding of the disease. Participants can now enroll at