Background
Rare Trait Swiss is a Swiss non-profit association, founded in March 2021 by the parents of a child affected by this rare lysosomal storage disease, aspartylglucosaminuria (AGU). AGU is a progressive degenerative disease that kills affected children as early as adulthood, aged 25 to 35 years. There are only about 200 known cases of this disease worldwide. More details about the disease can be found on our website (www.raretraitswiss.ch).
Rare Trait Swiss is an independent, non-political association whose nature is to work for; the charity of patients with Aspartylglucosaminuria (AGU), information for families of patients and healthcare professionals who want to know more about (AGU).
Aspartylglucosaminuria (AGU) is one of the rare lysosomal diseases, very little understood, for the time being a cure still remains to be developed and administered to humans.
The association works together with Rare Trait Hope Fund, a 501(c)(3) registered charity in the United States and Rare Trait Hope Society registered in Vancouver Canada to raise money to fund expenses associated with the preparation for and conduct of an upcoming gene therapy clinical trial. Together, we need to raise US$2 million and we hope to raise CHF500,000 of that here in Switzerland
Thanks to the combined efforts of parents of affected children in the USA, France, and Canada and the academic work of professors, researchers, geneticists, and other scientists, important advances have been made in gene therapy that suggests a future a very near cure for affected patients.
No research to develop a cure for AGU had been conducted anywhere in the world before 2012. Parents of two children with UGA located in the United States organized and funded private research at the University from North Carolina. The aim of the research was to develop a cure for UGA.
Based on the efforts of these two parents, many activities were completed including animal study, safety study, partial completion of natural history study in humans, and deposit of a pre-IND (initial new drug request) to the FDA (Food & Drug administration) in the USA. The organization of a clinical trial in humans is underway. Due to the extreme rarity of the disease, funding this trial is a real challenge for families of AGU patients.
Rare Trait Swiss wishes to combine its efforts with those of associations such as Rare Trait USA , Rare Trait Hope in Canada and “Vaincre l’AGU” in France which are working to facilitate the remaining research and development work necessary to treat and cure AGU. The funds raised will support the information of this very little known disease and the funding of the clinical trial. We will seek financial contributions for this including, but not limited to, individuals, nonprofits, government agencies, and businesses.
