WESTPORT, Conn., Sept. 18, 2018 /PRNewswire/ -- Coalition to Cure Calpain 3 (C3), a non-profit patient advocacy organization
"Gene therapy has shown tremendous promise in numerous diseases caused by a single malfunctioning gene, including in more common forms of muscular dystrophy and other LGMD subtypes," said Jennifer R. Levy, PhD, scientific director at C3. "Because LGMD2A is caused by a malfunctioning calpain 3 gene, we believe that gene therapy holds great promise to treat this debilitating disease for which there is currently no treatment or cure. Our gene therapy initiative is designed to study a number of gene therapy techniques with the goal of advancing potential treatments toward the clinic as quickly as possible."
C3 recently funded two new research grants to explore the potential of gene therapy for LGMD2A :
These grants mark the third and fourth research grants, respectively, that C3 has funded as part of its gene therapy initiative, with a commitment to date of more than $500,000.
In 2017, when this initiative was launched, C3 funded two gene therapy projects:
"As our Gene Therapy Initiative research projects near their first milestones, we are excited to soon learn which approaches have the potential to ultimately benefit patients with this serious disease," says Dr. Levy.
Limb-girdle muscular dystrophy type 2A (LGMD2A), which is also called calpainopathy, is a progressive muscle-wasting disease caused by defects in the calpain 3 gene. LGMD2A is a rare disease and the most common single form of limb-girdle muscular dystrophy (LGMD), representing an estimated 30 percent of all LGMD cases. It is estimated that LGMD2A affects roughly one in every 43,000 people.
In most cases, LGMD2A patients experience symptoms by age 20. Roughly half of LGMD2A patients experience muscle contractures, which may cause toe-walking and scoliosis. LGMD2A affects large muscles most and results in both weakness and reduced exercise endurance. Eventually, patients have difficulty with daily living activities such as climbing stairs, rising from a chair, or getting up off of the floor. LGMD2A patients typically lose their ability to walk within 10 to 30 years from the first onset of symptoms. Unlike some other forms of muscular dystrophy, heart and lung involvement is fortunately rare, and life expectancy may be near normal.
About Coalition to Cure Calpain 3
Coalition to Cure Calpain 3 (C3), a national, public, 501(c)(3) non-profit organization, was founded in 2010 for the specific purpose of funding research efforts focused on understanding the biology of and finding a cure for LGMD2A/calpainopathy. C3 was created by people with LGMD2A for people with LGMD2A, as both C3 founders have this progressive disease. C3 is motivated by its desire to encourage collaboration among scientists, those who have LGMD2A, their families, friends and the community at large to bring an end to this under-researched, underfunded rare disease. For more information, visit http://www.curecalpain3.org.
Contact:Jennifer R. Levy, PhDScientific Director, C3(319)email@example.com
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SOURCE Coalition to Cure Calpain 3
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