A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Conditions
Pompe Disease (Late-onset) · Pompe Disease Late-Onset · LOPD
Simple summaryAI-assisted
This summary is produced by a language model and is experimental. It may be incomplete or inaccurate and is not a substitute for professional medical advice.
Locations
Barrow Neurological Institute, Phoenix, Arizona, United States
Contact: Principal Investigator (Shafeeq Ladha, MD)University of California, Irvine (UCI), Irvine, California, United States
Contact: Principal Investigator (Tahseen Mozaffar, MD)Stanford Neuroscience Health Center, Palo Alto, California, United States
Contact: Principal Investigator (John Day, MD, PhD)NYU Langone, New York, New York, United States
Contact: Principal Investigator (Nicolas Abreu, MD)Duke University, Durham, North Carolina, United States
Contact: Principal Investigator (Natalie Katz, MD, PhD)Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, United States
Contact: Principal Investigator (Paul McIntosh, MD)University of Pittsburgh Medical Center (UPMC), Pittsburgh, Pennsylvania, United States
Contact: Principal Investigator (Paula Clemens, MD)University of Texas Southwest Medical Center, Dallas, Texas, United States
Contact: Study coordinator (Markey McNutt, MD, PhD) · geneticsresearch@utsouthwestern.edu · 214-645-7411Contact: Principal Investigator (Markey McNutt, MD, PhD)Virginia Commonwealth University (VCU), Richmond, Virginia, United States
Contact: Principal Investigator (Susan Sparks, MD, PhD)