Our families matter, we believe that gene therapy and other treatments are within reach, we believe that the only way to succeed is together.
Our vision is a cure, our mission is connecting people and collaborating to achieve a cure. This involves education, patient advocacy and facilitating research activities that aim to cure PHTS. Our research focus areas are our patient registry, future development of cell lines, and fundraising to encourage pre-clinical research.
You can make a difference. Help us raise one million dollars by the end of 2019. Every dollar earned through the campaign will go to research. All you have to do is donate a minimum of $1 and share this post with one reason why this campaign matters for you or your family.