Research & Drug Development
Developing a Treatment for B3GALNT2-Related CMD
Our groundbreaking research focuses on gene therapy approaches to treat B3GALNT2-related Congenital Muscular Dystrophy. While this is just one of the mutations causing CMD, our research will drive progress for every patient and family fighting CMD.
Meet Our Research Team
Qi Long Lu, MD, PhD
Qi Long Lu, MD, PhD. Dr Lu graduated from Shanghai Second Medical University, Shanghai, China and obtained PhD degree at University of London, England, UK. Dr Lu is the Professor at Department of Orthopaedic Surgery, Wake forest University and Atrium health, and has served as the Director at McColl Lockwood Laboratory for Muscular dystrophy Research since the establishment of the laboratory. Dr. Lu ’s main research interests are experimental therapies for Duchenne and Limb Girdle muscular dystrophies, including gene therapy, oligonucleotide therapies and drug development.
Anthony Blaeser, PhD
Anthony Blaeser, PhD is a Research Assistant Professor and Associate Director of the McColl-Lockwood Muscular Dystrophy Lab at the Atrium Health Musculoskeletal Institute. He received his M.S. in biotechnology and PhD in cellular and molecular biology from Illinois State University where his research focuses were on endocrinology and the effects of aging on immune system function, respectively. Dr. Blaeser continued work at Illinois State University as an Instructional Assistant Professor teaching graduate and undergraduate students. He joined the McColl-Lockwood lab in 2011 examining disease progression in a mouse model for FKRP-related muscular dystrophy. His work has been in characterizing the FKRP mutant mouse models, examining age- and development-associated effects on muscular dystrophies, and developing therapeutics for muscular dystrophy including drug and gene therapies.
Development Milestones
Our drug development program is focused on gene therapy approaches for B3GALNT2-Related Congenital Muscular Dystrophy. Learn about our progress below.
In this foundational stage, leading researchers develop treatment prototypes to target the genetic root cause of B3GALNT2-Related CMD.
This stage involves rigorous testing in cellular and animal models to determine the optimal dosage and measure safety of a potential therapeutic.
Next, a successful treatment prototype must be "scaled up" into a medical-grade product. Gene therapy requires highly specialized facilities to produce "batches" of the treatment that are pure, stable, and consistent.
This is the final hurdle where the treatment is administered to patients under strict medical supervision. Divided into multiple sub-phases, these trials evaluate how the human body responds to the therapy.
Frequently Asked Questions
Support our work
Fighting Congenital Muscular Dystrophy
FightCMD, a non-profit organization (Tax ID number 41-2756694), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.