C3 News

Sarepta Therapeutics Q1 Update on LGMD Programs

Sarepta Therapeutics recently published a Community Letter to share updates on their limb-girdle muscular dystrophy gene therapy programs. Click here to access the letter on Sarepta’s website.

Join Coalition to Cure Calpain 3 in celebrating Rare Disease Day!

Rare Disease Day takes place on the last day of February every year, with the goal of raising awareness about rare diseases and their impact on patients’ lives. Over 300 million people are living with one or more of over

What’s on the Horizon for LGMD2A/R1?

Dr. Jennifer Levy, Coalition to Cure Calpain 3 Scientific Director, is a proud contributor to the Winter 2022 issue of LGMD News. This magazine, a project of The Speak Foundation, publishes up-to-date information for the LGMD community. Dr. Levy’s article

C3 to Participate in Externally-Led PFDD Meeting with FDA

If you are living with LGMD2A/R1, you will have the opportunity to participate in an externally-led Patient-Focused Drug Development Meeting (EL-PFDD) with the FDA on September 23, 2022. C3 is proud to be one of six LGMD organizations to collaborate

Carol Abraham appointed to Patient Engagement Collaborative

Today, the U.S. Food and Drug Administration (FDA), in collaboration with the Clinical Trials Transformation Initiative (CTTI), announced that Carol Abraham has been appointed to the Patient Engagement Collaborative (PEC) and will serve a two year term. Carol is the

A Special Message from Lionel Messi to the C3 Community

C3 is thrilled to share a special video message from soccer great Lionel Messi to promote September 30th as Limb-Girdle Muscular Dystrophy Awareness Day! CLICK HERE to donate to C3 to drive research for a cure, or text CALPAIN to

Together we are STRONGER: 7th Annual LGMD Awareness Day is coming up!

There is great strength in awareness, understanding, and unity. The LGMD community demonstrates global connectivity each year when we campaign together and gain recognition for LGMD. With over 30 different genetic types of LGMD, the act of collaborating globally makes

Register now for the 2021 International LGMD Conference

Are you going to the 2021 International LGMD Conference? It should be an amazing event and C3 is looking forward to participating. C3 Scientific Director Dr. Jennifer Levy will moderate a group discussion and C3 Director of Community Outreach Carol

Sarepta announces gene therapy program for LGMD2A/R1

Today, Sarepta Therapeutics, Inc. announced that it executed an exclusive license agreement for an LGMD2A/R1 gene therapy program developed by Dr. Zarife Sahenk at Nationwide Children’s Hospital. Preclinical research conducted by Dr. Sahenk provided early proof of concept data for

FUNDING OPPORTUNITY! Coalition to Cure Calpain 3 Issues Request for Applications for Research Related to LGMD2A/R1

Coalition to Cure Calpain 3 requests applications for research and translational projects related to calpain 3 and limb-girdle muscular dystrophy type 2A (LGMD2A, also called LGMDR1 Calpain 3-related). The goal of the RFA is to enable the development of therapeutic