News Releases
Golden Heart Flower Granted FDA Orphan Drug Designation for GHF-201 for the treatment of Glycogen Storage Diseases Type IV, including APBD
We are excited to share that Golden Heart Flower, Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s product GHF-201 to treat glycogen storage disease type IV, including APBD. Golden Heart [...]
APBD Featured in New Book, Kaleidoscope: Rare Disease Stories
Kerry Wong We are excited to share that Sarah William’s APBD journey is featured in the newly published book, Kaleidoscope: Rare Disease Stories, by Kerry Wong. The stories featured in this book are firsthand narratives from individuals [...]
APBD Community Shines Bright on Rare Disease Day
The APBD community joined the National Organization for Rare Disorders and rare disease communities around the world to celebrate Rare Disease Day on February 29th. Leading up to the day, we urged our community to wear [...]
Duke University and Research Fund of the Hadassah Medical Organization Scientists Awarded Million Dollar Bike Ride Grants for APBD Research
The APBD Research Foundation is pleased to share that Or Kakhlon, PhD (Research Fund of the Hadassah Medical Organization) and Priya Kishnani, MD, MBBS and Rebecca Koch, PhD, RDN, LDN (Duke University Medical Center) are the recipients of rare disease [...]
Golden Heart Flower Granted FDA Orphan Drug Designation for GHF-201 for the treatment of Glycogen Storage Diseases Type IV, including APBD
We are excited to share that Golden Heart Flower, Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s product GHF-201 to treat glycogen storage disease type IV, including APBD. Golden Heart [...]
APBD Research Foundation Expands its Scientific and Medical Advisory Board with the Addition of Dr. Jennifer Orthmann-Murphy, Dr. Felix Nitschke, Dr. Matthew Gentry, and Dr. Wyatt Yue
The APBD Foundation is excited to announce key additions to its Scientific and Medical Advisory Board (SMAB). From left to right: Jennifer Orthmann-Murphy, MD, PhD; Felix Nitschke, PhD; Matthew Gentry, PhD; and Wyatt Yue, PhD [...]