For Patients

 

Tell your story and make a difference by joining the NEW patient-powered LGMD2A/Calpainopathy Registry

 

C3 IS EXCITED TO ANNOUNCE OUR NEW LGMD2A/CALPAINOPATHY REGISTRY. The registry will improve our understanding of the symptoms of Calpainopathy (including LGMD2A/R1 and LGMD1I/D4) and how they change over time. The study will also allow participants, if they choose, to be contacted about research studies and clinical trials. Patient-entered data is collected through a secure web-based application developed and maintained by the National Organization for Rare Disorders (NORD®).

The purpose of the LGMD2A/Calpainopathy Registry is to bring the Calpainopathy community together and collect data that is an essential requirement for policy makers, academic researchers, and therapeutics companies to advance treatments for the disease. Some of the goals of the Calpainopathy Patient Registry are:

• To describe the people who have LGMD2A/Calpainopathy and to better understand the stages of the disease and the different ways the disease affects people.
• To understand how LGMD2A/Calpainopathy changes over a person’s lifetime.
• To help to develop management guidelines to improve the care and quality of life for people with LGMD2A/Calpainopathy.
• To identify people with LGMD2A/Calpainopathy who might be willing to take part in other research studies or clinical trials.

How can you help drive research and speed development of a treatment?

If you or your child is living with Calpainopathy, join the registry today at LGMD2A.IAMRARE.org.

For more information, see our Frequently Asked Questions document or contact us at Registry@CureCalpain3.org.

 

FAQ for patients and families

Coalition to Cure Calpain 3 has published a list of answers to Frequently Asked Questions about LGMD2A (a form of calpainopathy). You may download the FAQ here.

 

Clinical research opportunities for individuals with LGMD2A

Coalition to Cure Calpain 3 maintains a list of studies that are recruiting individuals with LGMD2A. Click here to learn more about clinical research opportunities.

 

Resources

• NIH: Genetics Home Reference LGMD
• NIH Description of LGMD2A
• NIH GeneReviews – Calpainopathy
• NIH: The Calpain 3 (CAPN3) Gene
• Muscular Dystrophy Association