Finding Hope for FRRS1L strives to share information and resources with the public, medical professionals, and researchers to increase the awareness and understanding of FRRS1L.

PRESS RELEASES

  • August 24, 2026 - Finding Hope for FRRS1L and QUATRE LAB Partner to Advance Gene Therapy Development for Children Living with FRRS1L Disease

    Today, Finding Hope for FRRS1L is proud to announce a new collaboration with QUATRE LAB, a specialized Contract Research Organization (CRO) focused on Advanced Therapy Medicinal Products (ATMPs). This partnership brings additional world-class scientific and analytical expertise to the organization's gene therapy program and represents another important milestone as the program advances toward first-in-human clinical evaluation.

  • June 30, 2026 - Finding Hope for Frizzle (FRRS1L) Granted Rare Pediatric Disease Designation by the U.S. FDA for Investigational Gene Therapy

    Finding Hope for Frizzle (FRRS1L), a parent-led nonprofit organization dedicated to developing treatments for children living with FRRS1L-related developmental and epileptic encephalopathy (DEE37), today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to the organization's investigational gene therapy program, TfR1 CapX/FRRS1L.

  • May 8, 2026 - Finding Hope for Frizzle (FRRS1L) Announces U.S. FDA Orphan Drug Designation for FRRS1L Gene Therapy

    Finding Hope for Frizzle (FRRS1L) today announced a significant milestone in its mission to develop a treatment for Frizzle (FRRS1L) genetic disease. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for FRRS1L/TfR1 CapX, Frizzle gene replacement therapy, for treatment of developmental and epileptic encephalopathy type 37 (DEE 37).

  • February 26, 2026 - Finding Hope for FRRS1L Selects Parexel for Regulatory and Investigational New Drug (IND) Support with US Food and Drug Administration (FDA)

  • February 18, 2026 - Finding Hope for Frizzle (FRRS1L) and Apertura Gene Therapy Announce License Agreement for the Development of a Gene Therapy Using TfR1 CapX, an AAV Capsid Designed to Target the Central Nervous System

  • February 18, 2026 - Finding Hope for Frizzle (FRRS1L) Announces Strategic Partnership with Viralgen for FRRS1L Gene Therapy Manufacturing

Check out our latest updates on our Instagram page: @Hope4FRRS1L

View fullsize #didyouknow Frizzle treatment will be gene replacement therapy that will be a one time dose? Learn more at our new section on our website labeled VIDEOS! Watch a video developed by @asgctherapy called “Gene Therapy Basics” #frrs1l #geneth
View fullsize So thankful this Frizzle story is getting picked up in so many places! We hope it helps more #FRRS1L families find our organization and register! We also hope it lands on the hearts of more caring people who want to join us in changing lives and chan
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View fullsize #didyouknow we have no paid staff and 97% percent of dollars raised in 2025 went directly to pay for treatment development? When donors give to @hope4frrs1l they know their dollars are going to change lives and #changetheworld #frrs1l
View fullsize #thankyou @denverpost for helping spread awareness of the devastating genetic disease #frrs1l but also our hope to develop lifesaving treatment by next year! #denverpost #news #fortcollins #denver
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View fullsize Help us reach our $500,000 year end goal! So far we have raised $149,000. Thank you to all who have helped us so far! All dollars raised will go to funding the manufacturing of the treatment next year! Please continue to share our story of Hope! #end
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FRRS1L in the News

  • A young girl with glasses and a headband smiling and looking at the camera. She appears to be in an indoor setting.

    Daily Mail

    Girl, 8, hit by rare genetic condition that has left her trapped in her body and only able to communicate with her eyes... but big pharma says it's too expensive to develop drug to help her

    Click Here
  • Front page of a newspaper titled 'The Sunday Denver Post' dated December 28, 2025, featuring a headline 'Brotherly Love' and a photo of three children, one lying in a hospital bed with a book, wearing glasses, and two others leaning over her, with an announcement thanking for sharing the story about Frizzle disease.

    Denver Post:

    A Fort Collins family is trying to raise millions to test gene therapy that could help kids trapped in bodies they can’t move

    Producing a treatment for 8-year-old Everly Green’s ultra-rare genetic condition isn’t profitable for drugmakers

    Click Here
  • A graphic with a speech bubble containing the text 'THANKS for doing that!' and the phrase 'episode 4' below. The name 'CHRISSY GREEN' appears at the bottom in bold.

    Thanks for Doing That Podcast: FRRS1L

    In this special release, Heather talks with Chrissy Green about the reality for kids living with FRRS1L . Chrissy dives into the biology and science in the episode; but, basically the FRRS1L disorder causes disruption to messages in the brain and affects life holistically for these children and their families.

    Finding Hope for FRRS1L is a parent initiated non profit that is seeking to foster community, create awareness, and find a cure. They have had ground breaking results in trial studies and are moving forward to get the treatment to the children.
    Listen Here
  • Family photo of Gladys Soto, Frank Diaz, and their four children in a living room. The children are Zania, Dioan, Ian, and Alec, with two of them in wheelchairs. The family is smiling and posing for the picture.

    Observer Today - Dunkirk family seeks help for rare genetic disorder

    A feature story on Zania and Alec, two FRRS1L kids that are brother and sister, and the hope to get a cure if the funds can be raised to pay for it.

    Read Here
  • A family sitting outdoors amid fall leaves. The mother and father are holding their two young children, one of whom has a bow in her hair and is looking up, while the other, a toddler, is sitting on the mother's lap. All are dressed in casual autumn clothing and smiling.

    Oregonian - Saving Providence

    A feature story on saving Providence who has a life debilitating genetic disorder called FRRS1L.

    Read Here

  • North Forty News- Colorado

    Fort Collins Family Raising $400,000 to Develop Treatment for Genetic Disorder.

    Read Here

  • Despierta America, Univision USA

    Media coverage about the story of Arturo's journey with FRRS1L and the hope for a cure, along with commentary from Dr Xilma Ortiz-Gonzalez from the Children’s Hospital of Philadelphia about the founder mutation effect in children of Puerto Rican heritage.

    Watch Here

  • El Nuevo Dia, Puerto Rico

    Media coverage about the story of Arturo's journey with FRRS1L and the hope for a cure, along with commentary from Dr Xilma Ortiz-Gonzalez from the Children’s Hospital of Philadelphia about the founder mutation effect in children of Puerto Rican heritage.

    Watch Here

  • WAPA TV, Puerto Rico

    Media coverage about the story of Arturo's journey with FRRS1L and the hope for a cure, along with commentary from Dr Xilma Ortiz-Gonzalez from the Children’s Hospital of Philadelphia about the founder mutation effect in children of Puerto Rican heritage.

    Watch Here

Contact us.

We would love to hear from you. For feedback, comments, or questions, please use the contact us form.


 

If you are the family of a child with FRRS1L, and are not yet connected with our cause, please fill out the Register form to receive updates on fundraising and the development of treatment.

General FRRS1L Facebook: Finding Hope for FRRS1L 

Family Support Group on Facebook link: FRRS1L Support Group

Instagram: @Hope4FRRS1L