C3’s 2018 Year in Review

Happy New Year! We at Coalition to Cure Calpain 3 (C3) thank our Board of Directors for working alongside us and our generous donors for helping us realize these achievements in 2018:

THREE NEW RESEARCH GRANTS AWARDED IN 2018

Development of Gene Therapies:

C3 awarded grants to Dr. Isabelle Richard, Genethon, to analyze a new model of limb girdle muscular dystrophy type 2A (LGMD2A, a form of calpainopathy) and utilize it in the development of adeno-associated viral (AAV)-mediated gene therapy, and to Dr. Zarife Sahenk, Nationwide Children’s Hospital, to test the safety and efficacy of AAV-mediated gene therapy in a mouse model of LGMD2A.

Testing Modulation of Mitochondria Activity in an LGMD2A Model:

Dr. Kathryn Wagner, The Johns Hopkins School of Medicine, is examining a method to improve mitochondrial activity as a potential therapeutic option for LGMD2A.

Learn more

 

C3 FOSTERS SCIENTIFIC COLLABORATION

C3 Scientific Director Dr. Jennifer Levy organized a conference focused around increasing our understanding of LGMD2A and preparing for future clinical trials in order to accelerate the development of a therapy for this disease. In attendance were prominent neuromuscular clinicians, world renowned research scientists from academia and industry, and representatives from various funding agencies.

Learn more

 

C3 MAINTAINS THE INTERNATIONAL LGMD2A REGISTRY

C3 is proud to have established the International LGMD2A Registry, which now has over 900 submissions!

This registry is important as it:
• Facilitates a better estimate of the number of people living with LGMD2A, and draws attention to the need for a treatment and cure.
• Allows researchers to better understand the progression and manifestations of the disease, and locates participants for clinical trials – when available.
• Helps with fundraising and awareness efforts.

Therefore, we encourage you or anyone you know who is living with LGMD2A to register. This is an international database open to patients living in ALL countries and is open to ALL ages.

Visit the LGMD2A Global Patient Registry

 

C3 LAUNCHED A GENE THERAPY INITIATIVE

C3 is accelerating the understanding of the potential for gene therapy to treat LGMD2A by funding research projects testing several approaches. Over $500,000 has been committed to the four grants that have been awarded as part of this initiative.

Donate to the Gene Therapy Initiative

 

C3 BRINGS TOGETHER FAMILIES IMPACTED BY THIS MUSCLE WASTING DISEASE

To learn more about this disease and/or connect with others who are living with its effects, we suggest that you:

• Subscribe to the C3 NEWS PAGE which is frequently updated with C3 happenings
• Join the LGMD2A GLOBAL PATIENT REGISTRY
• Encourage friends and family to SUBSCRIBE TO OUR EMAILS
• “Like” our FACEBOOK PAGE
• JOIN THE “C3 COMMUNITY”, our closed Facebook group for patients and their families to share insights, advice, and support

 

C3 IS DEDICATED TO RAISING AWARENESS ABOUT LGMD2A

C3 joined organizations representing multiple LGMD subtypes to draw attention to LGMDs on September 30, LGMD Awareness Day. Spearheaded by C3 Director of Community Outreach Carol Abraham, initiatives included:

• “Lime Green for LGMD,” where individuals wear lime green clothing and wristbands
• LIMEmoji Challenge: re-create your favorite Emoji using a lime, and challenge your friends and family to do the same
• State, city, and county proclamations of September 30 as Limb Girdle Muscular Dystrophy Awareness Day
• Social media campaigns on Twitter and Facebook
• LGMD interviews and resources available at LGMD-Info.org

Want to be involved in the next LGMD Awareness Day? Volunteer here!

 

HELP US FIND A CURE!

The best thing you can do to help C3 find a cure is to donate. To make a donation, please:

• VISIT OUR SITE to donate via credit card OR
• MAIL THIS FORM to pay by check