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. This appointment formalizes the work she has undertaken for many years as the parent of a teenager living with LGMD2A/R1.
Melina has lent her voice advocating for our community in myriad ways, including:
- LGMD Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting – Melina joined her daughter Brooklyn, who is living with LGMD2A/R1, to candidly discuss the burdens of this disorder on their family at a meeting organized by a group of LGMD non-profits that included C3. The goal of the subsequent meeting report is to provide the U.S. Food and Drug Administration (FDA), clinicians, medical product developers, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment.
- LGMD2A/R1 Standards of Care Workshop – Melina served as a patient representative at this meeting, organized by Dr. Volker Straub, Director of the John Walton Muscular Dystrophy Research Centre at Newcastle University, and funded in part by C3. LGMD2A/R1 clinical experts, patients, and advocates convened to deliberate and develop care standards for implementation in clinical practice globally. The publication of the SOC will benefit patients by educating healthcare providers about this rare form of muscular dystrophy. SOC guidelines are often a prerequisite to allow for the fair comparison of outcome measures in multi-center and international clinical trials.
- LGMD Day on the Hill – Melina has taken part in this advocacy initiative, organized by our colleagues at The Speak Foundation, for two years. She shared this recap about her experience last fall, “I had the honor of joining my LGMD family in Washington, D.C. to raise our voices for everyone living with Limb-Girdle Muscular Dystrophy. We met with lawmakers, shared our stories, and pushed for policies like the Rare Pediatric Disease Program that can accelerate research and improve quality of life for all who face LGMD. Every voice matters, and together we are making sure LGMD is seen, heard, and prioritized.”
- LGMD Awareness Day Facebook Fundraiser – For six consecutive years, Melina and her family have spent the month of September successfully building awareness of LGMD2A/R1 and raising critical dollars for C3-funded scientific research. Our organization has committed over $3 million to projects focused on calpainopathy.
In addition to these experiences, Melina can share personal perspectives as a caregiver to a patient participating in LGMD2A/R1 natural history studies and the LGMD2A/Calpainopathy Registry (where she served as a beta tester in the C3 development process). Looking ahead, she’ll keep us posted on policies and issues that impact our community and will represent C3, along with Dr. Jennifer Levy, Scientific Director, and Jordan Boslego, President, in the Patient Advocacy Pavilion at the MDA Clinical and Scientific Conference this March.