We believe that patients hearing “there’s nothing else to try,” is unacceptable.

Our focus is on developing disease-modifying medicines for glycogen storage diseases, lysosomal storage diseases and beyond.

As part of our efforts to search for ways to help a family member diagnosed with Adult Polyglucosan Body Disease (APBD), an adult-onset genetic disorder (GSD type IV), we decided to take action by establishing a new company called GHF-Golden Heart Flower*, aimed at promoting translationally-oriented research and development of drugs to treat APBD and other rare glycogen storage and lysosomal storage diseases with either limited or no foreseeable cure.

Our portfolio consists of several easily administered, innovative small molecules that represent new approaches to correct processes and clinical manifestations of such difficult-to-treat diseases.

Based on additional outcomes derived from our recent preclinical research, it appears that our pipeline compounds may provide new approaches for other medical conditions with high prevalence.

Golden Heart Flower is the name of a Hebrew short story, first published in 1930 by the children’s author Shlomo Zalman Ariel. This story tells of a child whose mother falls asleep and no one knows how to heal her. An elderly doctor explains to the child that the only way to save her is by finding a red flower with a heart of gold, that can only be found in the distant mountains. Once the mother smells it, she will recover. Of course, the boy finds the flower, and everyone lives happily ever after.