HOPE IN ACTION

Frizzle Fundraising Campaign to get a clinical trial started by September 2026

A young boy lying in a hospital bed, connected to medical monitors and equipment, with a healthcare professional standing nearby in a hospital room.
Close-up of a young child with an oxygen tube on their nose, wearing a hospital cap and colorful hospital gown, lying in a hospital bed.
Young boy sleeping in bed with a nasal oxygen mask attached to his face.
A young girl with dark hair wearing glasses is resting in a hospital bed. She has a breathing tube and is holding a Minnie Mouse plush toy. The background has colorful paper hearts taped to the wall.
A young girl with curly hair smiling while sitting in a wheelchair in a living room.
Young girl lying in a hospital bed with a medical IV attached, smiling and wearing a pink sleeveless shirt with colorful flower and unicorn prints, with a pink hair bow decorated with butterfly and star shapes, resting her head on a pink and blue pil
A young girl with dark curly hair sitting in a walker with a tray table. She is wearing a black and gray sensory vest and has a colorful toy on the tray. The background shows a living room with a brown sofa, a floor lamp, and a closed door. The word
Young man with disability sitting in a wheelchair with a head support cushion, surrounded by colorful toys and a mobility aid, in a living room with a brown couch and window blinds.
Young girl in a pink and black wheelchair being examined by a medical professional. The girl has glasses and looks slightly unwell. The room has various medical and craft supplies.
Young boy in a wheelchair at a therapy or medical facility, wearing a gray Nike shirt and a specialized blue harness or vest for support, with a nasal oxygen tube, in a room with colorful tiles and exercise equipment in the background.

2026 the Year for Frizzle Treatment

But only with your support!

  • Our pre-clinical research on Frizzle (FRRS1L) mouse models showed the restoration of brain and motor function through gene replacement therapy. This research leads us to hope and believe Frizzle gene therapy treatment will bring patients back to function!

  • Our goal is to begin treating Frizzle patients by September of this year with gene replacement therapy given as a one-time dose.

  • We have a clear plan, committed partners, and a defined timeline. What remains is raising the funds needed to bring this treatment across the finish line and into patients.

  • We must raise $4 million to cover both the manufacturing of the drug and the costs of treating patients in a clinical trial.

  • Every donation goes directly toward changing lives and changing the world!

THANK YOU!