Over the past two days, I had the privilege of representing Coalition to Cure Calpain 3 in my advocacy chair role at LGMD Policy Action Day on Capitol Hill. Our community came together for a full schedule of meetings across both the House and Senate, ensuring that the realities of ultra-rare drug development remain front…
Read MoreNew Year, New Chair: C3 Welcomes Melina Garza to New Role as Advocacy Chair
C3 is thrilled to share that Melina Garza is stepping into a newly-created volunteer position. As Advocacy Chair, Ms. Garza will represent our organization educating stakeholders about calpainopathy and engaging them in C3’s mission to drive research toward a treatment or cure.
Read MoreC3 Attends the 2025 International LGMD Conference
C3 was proud to be a part of the International LGMD Conference which convened July 18-20 in Orlando, Florida. This event is hosted by the Speak Foundation and focuses on all subtypes of LGMD. Among the highlights:
Read MoreLGMD2A/R1 Standards of Care Workshop
Coalition to Cure Calpain 3 (C3) is proud to have partnered with the John Walton Muscular Dystrophy Research Centre, Sarepta Therapeutics, the Speak Foundation, and LGMD Awareness Foundation to sponsor the LGMD2A/R1 Standards of Care (SOC) Workshop. This meeting, organized by Dr. Volker Straub, Director of the John Walton Muscular Dystrophy Research Centre at Newcastle…
Read MoreKNOW YOUR CODE: Why the new ICD-10 codes matter, and what you can do to confirm your records
Did you know that new ICD-10 codes were assigned for limb-girdle muscular dystrophies (LGMDs) in October 2022? Patients with LGMD2A/R1, autosomal recessive LGMD due to Calpain 3-dysfunction, have been given a diagnosis code of G71.032. Patients with LGMD1i/D4, autosomal dominant LGMD due to Calpain 3-dysfunction, are grouped with other autosomal dominant LGMDs with a diagnosis…
Read MoreNew Report Highlights Unmet Medical Needs of Six Subtypes of LGMD, Including LGMD2A/R1
Today, Coalition to Cure Calpain 3 joins five other LGMD patient organizations (collectively referred to as The LGMD Coalition) to release the ‘Voice of the Patient Report’ summarizing outcomes from the September 2022 Externally-Led Patient Focused Drug Development Meeting (EL-PFDD).
Read MoreC3 partners with 16 LGMD organizations in letter to FDA
Coalition to Cure Calpain 3 is proud to be one of 17 organizations dedicated to LGMD to have collaborated on a statement submitted to the Food and Drug Administration in conjunction with the recent Patient-Focused Drug Development Meeting Patient Perspectives on Gene Therapy Products. Special thanks to The Speak Foundation, Joshua Thayer Esq., and Bradley Williams…
Read MoreFDA Patient Listening Session on LGMDs held October 20, 2020
On October 20th, 2020, the LGMD community was able to meet with the FDA to share our patient experiences. This was a patient-led listening session organized by a consortium of advocacy organizations, including Coalition to Cure Calpain 3. The session focused on various aspects and genetic subtypes of LGMD. Fifteen presenters, all patients or family…
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