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 therapy, and are often longitudinal, meaning that they collect information from the same person at different timepoints to assess symptom progression.
Why are natural history studies important?
Natural history studies play a critical role in the development of therapies. They help illuminate the clinical features, symptoms, and progression of diseases, which is especially important in the case of an ultra-rare disease such as LGMD2A/R1 (Calpainopathy). This information is used to design clinical trials that will test future therapies.
Why participate in a natural history study?
Many people enjoy participating in these studies. In addition to contributing to valuable research, participants often meet LGMD experts with extensive knowledge about LGMD2A/R1. In the future, your study doctor may be able to help you identify clinical trials in which you are eligible to participate.
It takes more people than you might think to make a research study happen!
Pictured here are the nearly 200 doctors, physical therapists, clinical research coordinators, and additional research staff who meet annually at the GRASP-LGMD Investigator Meeting to discuss the progress of their research, share data, and plan for the future. The LGMD patient community is also part of this team effort when participating in these research studies.
PLEASE NOTE: ENROLLMENT FOR GRASP-01-003 CLOSES APRIL 30, 2026
