This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
- Investigators
- Carla Zingariello, Sub Subramony
- Status
- Accepting Candidates
- Ages
- 12 Years - 50 Years
- Sexes
- All
Neuromuscular Medicine Specialist, Pediatric Neurologist, Pediatric Neuromuscular Rehabilitation Specialist
Dr. Zingariello is actively involved in research in her field. She is currently the Principal Investigator on a muscular dystrophy grant from CDC to University of Florida. She also serves as PI for a Limb Girdle muscular dystrophy clinical trial. She has a Children’s Miracle Network grant to investigate factors contributing to delays in diagnosis of juvenile myasthenia gravis and spinal muscular atrophy. She additionally serves as a sub-Investigator on multiple gene therapy clinical trials for Duchenne Muscular Dystrophy.
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the…
13 publications
2022
Developmental medicine and child neurology
•2022
Annals of clinical and translational neurology
•2022
Pediatric neurology
•2021
Child neurology open
•2020
Pediatric neurology
•