HOPE IN ACTION

An Evening for Frizzle

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SAVE THE DATE

  • Wednesday, May 20, 2026

  • 6:30pm-9:30pm

  • Rooftop, Macquarie Group Limited

  • 660 Fifth Avenue, New York, NY, 10103 US

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Please save the date for a special evening bringing together friends, partners, and supporters of Finding Hope for Frizzle (FRRS1L).

This event will support the final funding needed to complete manufacturing and launch the first clinical trial for children with Frizzle disease, with patient dosing planned for September 2026. Join us to learn more about the progress made, the path ahead, and how this community can help bring a potential first treatment to children and families who urgently need it.

Invitation to follow in April.

A smiling girl with curly hair in a pink shirt sitting in a wheelchair in a living room.
Young boy with curly hair sitting in a black stroller, wearing a green T-shirt with a yin-yang symbol and a black cap with a logo, making a relaxed facial expression.
Young girl lying in a hospital bed, connected to medical tubes and equipment, resting her head on a pink and blue bed pillow with a colorful blanket.

HELP FUND THE CURE

These sweet, but struggling children, now have HOPE that treatment is possible that will save their lives and change the world, but we need your help to fund it.

A young girl in a hospital bed wearing a patterned hospital gown, with medical tubes attached, a breathing mask, and a wrist band. She is resting with her eyes closed, surrounded by medical equipment and colorful toys.
A young child with curly hair wearing blue glasses and a bib that says 'Mini & Mighty' sitting at a table with a piece of bread.
A young girl with a serious expression wearing glasses, lying in a hospital bed with a breathing tube, holding a Minnie Mouse plush toy, surrounded by colorful paper hearts on the wall behind her.
A young boy with glasses and brown hair smiling, wearing a neck support, sitting in a wheelchair with an orange cover, in a room with curtains and a brick wall background.
Young girl in a wheelchair with a therapy vest and colorful shoes sitting at a glass table with a toy behind her. The image is taken indoors in a living room with furniture and a lamp.
A young boy with dark hair, smiling widely, sitting on a bed with blue and patterned pillows.
A young woman with long curly hair sitting in a wheelchair, holding a black object to her face, leaning slightly backward, with an open mouth and eyes half-closed, in a room near a sliding glass door with open blinds, sunlight streaming in.

2026 the Year for Frizzle Treatment

But only with your support!

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

  • 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!

Pre-clinical Data shows Restoration of Function

Our pre-clinical research on FRRS1L mouse models showed the recovery of function through gene replacement therapy. The gene replacement therapy given to FRRS1L diseased mice worked to bring function back to FRRS1L diseased mice brains and bodies! This is life changing news! We want recover for Frizzle patients and are working to get treatment developed this year. Would you join us in making treatment a reality for sick Frizzle patients this year?

Donate today to help us fund treatment development for Frizzle patients!

A female scientist wearing a white lab coat looking through a microscope in a laboratory with shelves of supplies and equipment.
A classroom or conference room with two women sitting at a table, watching a large screen displaying a close-up of a mouse on a white surface. There is a woman standing at a podium near a computer in the background.

Treatment by September 2026 is possible with your support!

Our race to treatment has begun. We have a goal to begin treating Frizzle patients by Sept. 2026, but it will only happen with your support! Donate today to help fund the manufacturing of the drug that will be a one time injection for Frizzle patients!

All donations are tax deductible, and go directly to fund the development of FRRS1L 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 diseases.

DONATE NOW