What can YOU do to drive LGMD2A/R1 research forward? Participate in a natural history study!

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.

There are multiple natural history studies enrolling individuals living with LGMD2A/R1.

Individuals living with LGMD2A/R1 are welcome to participate in multiple studies concurrently. Reimbursement for travel expenses may be available. Use the links below to find details about sites, enrollment criteria, and contact information.

JOURNEY Study

• Sponsored by Sarepta Therapeutics
• Multi-site international study
• Ages 4 years and older
• Enrolling both ambulatory and non-ambulatory individuals
• 3-year study

GRASP-003 Study

• Sponsored by Virginia Commonwealth University
• Multi-site international study
• Ages 12-50 years at time of enrollment
• Participants must be ambulatory
• 2-year study

LGMD2A/Calpainopathy Registry

• Sponsored by Coalition to Cure Calpain 3
• Collects natural history data through a web portal, with no travel involved
• Partipants can contribute data from anywhere in the world
• Open to anybody living with LGMD2A/R1 or LGMD1i/D4
• No anticipated date of termination