Double your donation on Giving Tuesday 2019

 

 

A “C3 Champion” has made a commitment to match every dollar donated on Giving Tuesday, up to $25,000.

Your contribution today to support scientific research for those families impacted by the muscle-wasting disease LGMD2A/R1 (a form of calpainopathy) will help us leverage these matching funds and bring us closer to a cure.

DONATE NOW

 

Read on to learn more about our achievements this year…

 

FUNDING HIGH POTENTIAL RESEARCH

C3 Awarded 2 New Research Grants in 2019

Dr. Nicholas Johnson, Virginia Commonwealth University: “Defining clinical endpoints in LGMD”

Establishment of an LGMD Research Network and development of tools necessary to test potential treatments in clinical trials

Dr. Jaakko Sarparanta, Folkhälsan Research Center & University of Helsinki: “Calpain 3-mediated proteolytic processing of C-terminal titin and turnover of titin fragments”

Investigating the physiological role of calpain 3 in cutting the protein titin, and how dysfunction of this activity may lead to muscle disease

Learn more about our research grants

C3 Awarded First Early Career Investigator Travel Award

Dr. Stefanie Müthel, Charité Medical Faculty and the Max-Delbrück-Center for Molecular Medicine: “Precise gene editing of LGMD2A/R1 causing mutations”

Development of a method to repair the mutation CAPN3c.550delA by using the gene editing technology CRISPR

Learn more about our early investigator travel awards

 

FOSTERING SCIENTIFIC COLLABORATION

Gene Therapy Workshop Focused on LGMD2A/R1

C3 Scientific Director Dr. Jennifer Levy organized a conference focused around understanding how best to facilitate the development and rigorous evaluation of candidate gene therapies for LGMD2A/R1. The workshop findings will guide grant reviews and associated investments in development up through an Investigational New Drug (IND) filing. The topics of the sessions included:

• Calpain 3 and LGMD2A/R1 clinical phenotype
• Regulatory perspectives
• Overview of a gene therapy program
• Discussion session 1: IND-enabling paths
• Discussion session 2: Clinical program readiness

In attendance were prominent researchers and clinicians, consultants with experience in biotech/pharma, and FDA staff members.

 

PROMOTING GLOBAL AWARENESS

Fifth Annual Limb Girdle Muscular Dystrophy Awareness Day was a Success

C3 joined organizations representing multiple limb girdle muscular dystrophy (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

Learn how you can be involved in the next LGMD Awareness Day

 

CONNECTING PATIENTS AND FAMILIES

• Visit and subscribe to the News page on our website. This page is frequently updated with C3 happenings.
• Join the LGMD2A Global Patient Registry
• Join our email distribution list
• “Like” our Facebook page
• Join the “C3 Community” our closed Facebook group for patients and their families to share insights, advice, and support

 

HELP US FIND A CURE!

Donate today and C3 will receive matching funds up to $25,000!