Finding Hope for FRRS1L serves to fund research and development to find a cure for FRRS1L gene disorder and other treatments that would improve the quality of life of children with FRRS1L.

Hope for a Cure

Witnessing a child lose abilities and independence is an indescribable pain. Currently, there are no medicines to control it, to slow it down, or to heal it. Parents are trapped in a hopeless inability to help their children, and there has been no viable prospect of finding treatment or a cure. Until NOW.


Recent studies of the FRRS1L gene have greatly advanced the understanding of the gene’s role in the body, and demonstrated that gene replacement therapy is not only possible for FRRS1L, but hopeful in the healing of our children. A team of gene therapists at the University of Texas (UT) Southwestern have agreed to partner with Finding Hope for FRRS1L to pursue the research and development of gene replacement therapy for FRRS1L children. This opportunity is life changing for FRRS1L children and families! Learn how you can help make this possible!

How to Help

  • DONATE

    DONATE to help us fund research and development for a cure for FRRS1L children. Note: all gifts are tax exempt.

    Your donation will fund the research and development of a gene therapy treatment. Your gift will help ALL children with FRRS1L. That is older children who have lost hope, younger children who haven’t yet regressed, and unborn children who will inherit this disorder. This research also moves forward the science that may result in cures for other genetic disorders.

    DONATE

  • SHARE

    SHARE our story to friends and family that would be willing to: support this cause, and increase awareness of FRRS1L.

    Our world is more connected than ever before through social media and email. Help us grow awareness of FRRS1L by sharing our story and posts on social media, and/or by writing an email sharing a link to our website. When you share our story you may help another FRRS1L family find us! In just a few months of starting our awareness initiatives we have already found five more families! You never know how the “share” button can change lives!

    SHARE

Gene Therapy

Currently we are pursuing the research and development of gene replacement therapy for FRRS1L gene disorder with Dr. Minassian at UT Southwestern. The team has been working with FRRS1L mice to prove that gene replacement therapy is not only possible, but it works on FRRS1L mice. We are at an exciting place with the research and are confident with funding and support, that we will reach clinical trials for FRRS1L children.

To learn more about gene therapy please check out the following resources: