by Madaline Spencer | Mar 5, 2024
In a significant development, Chiesi Global Rare Diseases has announced positive results from the Phase 3 BALANCE study, evaluating the efficacy, safety, and tolerability of pegunigalsidase alfa in adult patients with Fabry disease. Fabry disease is a...
by Madaline Spencer | Mar 4, 2024
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses recent clinical trials in Pompe disease. Pompe disease is a rare genetic, lysosomal disorder that causes progressive weakness in heart and skeletal muscles. It is...
by Madaline Spencer | Feb 29, 2024
Julia Alton, Executive Director at the Canadian Fabry Association, discusses the poster presented at WORLDSymposium 2024, “Exploring the experience of females living with Fabry disease in North America.” Fabry disease is a lysosomal storage disease...
by Madaline Spencer | Feb 15, 2024
Roger A. Levy, MD, Senior Global Medical Director, GSK Specialty Medicine, discusses Lupus nephritis, a rare kidney disorder. Lupus nephritis is a rare autoimmune, kidney disorder that is a complication of systemic lupus erythematous, commonly known as...
by Madaline Spencer | Feb 14, 2024
For many years, there has been a pressing need for effective treatment options for individuals living with Angelman syndrome. However, the recent Priority Medicine (PRIME) designation by the European Medicines Agency (EMA) for GTX-102 for the treatment of Angelman...
