The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.
Study Type
EXPANDED_ACCESS
Pegunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks).
University of Alabama-Birmingham
Birmingham, Alabama, United States
Phoenix Children's Hospital, Inc.
Phoenix, Arizona, United States
University of California Irvine
Orange, California, United States
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Central Coas Nephrology
Salinas, California, United States
University of Florida, Division of Pediatric Genetics
Jacksonville, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
University of Iowa
Iowa City, Iowa, United States
Infusion Associates
Grand Rapids, Michigan, United States
Dallas Nephrology Associates
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
...and 1 more locations