We are a PARENT-LED organization on MISSION to change the lives of ALL struggling Frizzle patients around the world!

We developed a GENE THERAPY DRUG for Frizzle Disease. Learn more HOW YOU CAN HELP fund a future clinical trial!

Learn more: TREAMENT for Frizzle
Learn more: WHY DONATE

QUICK INFO & LINKS

  • Friends and donors, thank you for caring about Frizzle kids! We have some helpful links to get you the informaiton you need:

  • Welcome! We’re so glad you found us. At Finding Hope for Frizzle (FRRS1L), we believe the knowledge gained through our own treatment development journey should be shared to help others move forward.

    We are committed to supporting parent/patient advocacy groups (PAGs) and rare disease organizations working to advance research and develop treatments for their communities.

    Explore our resources for practical information, lessons learned, and tools designed to help PAGs at every stage of the treatment development journey—from getting started to moving a potential therapy toward the clinic.

    • PAG resource page

  • Resources:

  • We have licensed Ben Deverman’s TfR1 CapX technology from Apertura Gene Therapy for our FRRS1L gene therapy program. We look forward to sharing insights that help move science and treatment development forward. Learn more about our desire to collaborate and share about CapX as our program develops.

  • At Finding Hope for Frizzle (FRRS1L), we are proud of what we have accomplished—as an organization, as parents of children living with Frizzle, and as drug developers working to move a treatment from an idea toward patients.

    We believe the lessons we have learned along the way can help strengthen the broader rare disease and drug development communities. Our team welcomes opportunities to speak, share our experience, and participate in industry conferences, panels, workshops, meetings, and educational events.

    Topics can include parent-led drug development, patient advocacy, treatment development, organizational ownership, partnerships, navigating the development process, and the unique perspective of being both parents and drug developers.

    Interested in having a member of our team speak at your event?

    Send your speaking request to contact@FRRS1L.org

  • Stay up to date with the latest Finding Hope for Frizzle (FRRS1L) organizational updates, treatment development news, partnerships, and announcements by visiting our Media Page.

    For media inquiries, interview requests, or additional information, please contact our team at contact@FRRS1L.org.

WHY THIS MATTERS 

CHANGE LIVES and CHANGE the WORLD for ALL NEURO DISEASE.

Finding Hope for Frizzle (FRRS1L) is a parent-led, Colorado registered 501c3 nonprofit organization, dedicated to developing life-changing treatment for children living with FRRS1L (pronounced Frizzle) genetic disease. The organization leads a comprehensive therapeutic development program spanning scientific research, regulatory strategy, manufacturing, clinical development, and patient advocacy, with the mission of bringing an effective gene replacement therapy treatment to patients as quickly as possible while helping advance the future of all neuro disease drug development. The organization has no paid staff and all donated dollars go to directly fund an upcoming FRRS1L clinical trial.

Finding Hope for Frizzle has licensed the new TfR1 CapX capsid technology. The collaboration marks a major milestone not only for families affected by Frizzle disease, but also for the future of gene therapy. The clinical program will utilize the innovative TfR1 CapX capsid, a next-generation AAV delivery platform designed to improve distribution of gene therapies throughout the brain following an intravenous (IV) infusion.

While a trial woud be focused on treating children with Frizzle disease, the implications with this therapeutic capsid extend far beyond a single rare disease. It has the potential to change gene therapy delivery for all neurological disease such as Alzheimer's and Parkinson’s disease. The successful clinical application of TfR1 CapX has the potential to accelerate the development of gene therapies for numerous neurological disorders by demonstrating a more efficient method of delivering genetic medicines throughout the central nervous system. If successful, the knowledge gained through this program could help shape the future of gene therapy for patients around the world. Learn more here.

WE NEED YOUR HELP!

images of Frizzle kids
A young girl with blonde hair lying in a hospital bed wearing a mask and pajamas with animal prints, holding a stuffed animal. Medical equipment and a remote control are nearby.
A young boy with glasses smiling and looking away, sitting in a wheelchair with a headrest.
A young boy with a disability sitting in a wheelchair, wearing a red Christmas-themed shirt and green pants. He has a medical headrest supporting his head and appears to be resting or sleeping.
A young girl with curly dark hair and light skin lying in bed with her mouth open. She is resting her head on a pillow with a star pattern, covered by a green quilt. There is a stuffed animal on her lap. The background shows curtains and a lamp on a
A young child lying in a hospital bed with a medical mask featuring ladybugs, connected to medical tubes and equipment, with an adult woman taking a selfie next to the bed.
A young boy in a wheelchair wearing a Mickey Mouse hat with big black ears, smiling and showing face paint that resembles whiskers, in a room with colorful artwork on the wall.
A young girl with braided hair, wearing a blue t-shirt with a graphic on it, sitting in a wheelchair, with a big blue bow in her hair and making a playful face.
A young girl with curly hair lying on a bed with blue sheets, wearing a black T-shirt with green and white text and graphics, and blue jeans. She is looking away from the camera.
Young boy lying in a hospital bed with a pacifier in his mouth and a bead necklace nearby, connected to medical equipment.
A young girl with glasses and a yellow bow in her hair lying on her side in a bed, wearing a yellow shirt with a Minnie Mouse print.
Young boy with a disability sitting in a specialized wheelchair with a tray and leg braces, holding colorful building blocks, near a window and plain wall.
A young girl in a pink dress and white bib sitting in a stroller near a body of water on a wooden dock, with trees and boats in the background.
Young boy in a wheelchair smiling with missing upper teeth, wearing a green shirt, and holding a white and pink toy dinosaur.
A young girl in a wheelchair wearing reindeer antlers and a striped dress sits in front of a decorated white Christmas tree, covered with blue and gold ornaments, with Christmas lights reflecting on the glass door beside her.
Young boy with glasses wearing a yellow patterned hat and a light yellow button-up shirt, sitting in a wheelchair indoors.
A young girl in a hospital bed smiling happily with a nasal cannula and medical equipment around her, wrapped in a colorful blanket.
A young boy with glasses sleeping in a wheelchair, wearing a plaid shirt, jeans, white sneakers, and a bib that says 'Grandma's Boy Gramma.'
A young person in a wheelchair wearing a Santa hat and glasses, with a hospital or care facility setting in the background and colorful balloons around
A girl lying in bed with a large stuffed teddy bear beside her, and a doll lying in her arms. The background shows a window with curtains and a view of the outdoors.
A young boy lying in a hospital bed with his eyes half-open, wearing a blue shirt. A person with a hospital wristband is gently touching or calming him, with their hand above his head. The bed has a metallic rail and a striped bedsheet, and the backg
A young girl with blonde hair and glasses smiling in a wheelchair, wearing a white shirt and colorful shorts, in a room with a pink balloon on the floor and various objects in the background.
A man wearing a red Santa hat, glasses, and a red shirt, smiling while sitting in a chair. A plush elf doll is perched on his shoulder, dressed in a Christmas outfit with a red hat, green suit, and red and white striped leggings.
A woman with long, curly hair wearing glasses and a striped shirt, appears to be surprised or in awe, lying on a bed with colorful rainbow and polka dot patterned pillows.
A young boy with curly brown hair and light skin sitting in a stroller, wearing a green T-shirt with a yin-yang symbol and a slogan, and a baseball cap with an outdoor adventure patch.
Woman with long curly dark hair in a wheelchair, wearing a red patterned top, blue bandana, and denim shorts, sitting indoors near a door with sunlight.
A young boy with brown hair and a beige sweater with a reindeer design lying on a hospital bed with a yellow pillow behind his head.
A young woman with dark, curly hair lying on a bed with white and floral bedding, yawning or singing, next to a stuffed white animal with black nose and eyes, in a bright room with a bedside lamp and white curtains.
A young boy with a medical tube in his nose sitting in a wheelchair at a hospital or clinic. He is wearing a gray Nike shirt and black shorts, with blue cushioned supports around his head, torso, and legs.
A young boy smiling in a stroller outdoors next to vibrant pink rhododendron flowers and green foliage.
Young person with dark hair styled in two buns, sitting in a wheelchair with a black headrest and armrests, wearing a yellow shirt and black pants, with eyes closed and mouth slightly open. In their lap is a black tray with an electronic device. The
Close-up of a young boy smiling, lying on a colorful, striped blanket with vivid pink, purple, green, yellow, and orange stripes.
Young girl in a wheelchair celebrating her 16th birthday, holding a sign that says "smart, strong, sweet 16." She is smiling with her eyes closed, in a bedroom with medical equipment in the background, colorful curtains, and a table with water bottle
A boy in a green shirt standing next to a boy in a wheelchair wearing a Santa hat, in front of a decorated Christmas tree with lights and ornaments. The boy in the wheelchair has a sad or tired expression, while the boy standing looks slightly smilin
A young girl with curly hair sitting in a wheelchair, smiling at the camera in a living room with a beige couch and patterned pillows.
Child in a ball pit filled with colorful plastic balls, looking up at the camera. The ball pit is in an indoor setting with a red, blue, and beige cushion backing, and items like a backpack and papers are visible on a surface in the background.
A young boy with glasses and brown hair sitting in a purple high chair, smiling and showing teeth, with a bandage on his nose, in an indoor setting.
Smiling children celebrating Christmas indoors near a decorated Christmas tree with presents, dressed in patterned pajamas, some with holiday accessories, with a partial view of a woman and festive decorations.
A young girl in a wheelchair wearing a red shirt and a necklace with medical tubes is looking up at Christmas decorations, including a reindeer-shaped balloon and a Santa Claus ornament.
A young boy with glasses sitting in a wheelchair, wearing orange protectors on his arms and legs, and a neck brace. He is inside a house near a door that leads outside, with a backyard visible through the glass door.
A young boy lying on a bed, wearing novelty sunglasses with the year '2023' on the frames and a 'Happy New Year' headband. The boy is wearing a T-shirt with cartoon characters on it.
A young boy with brown hair and closed eyes lying in a hospital bed, connected to a ventilator, with bandages on his wrist and ankle, with medical tubes attached.
A smiling young boy with short dark hair and braces, wearing an orange hoodie, lying on a bed with patterned pillows and blue sheets.
A young boy lying in a hospital bed with medical monitors and equipment around him, a healthcare worker attending to him.
A young man with glasses sitting in a wheelchair in a living room. He has a neck pillow with cartoon characters, and various toys hang on a white frame behind him. The room has a brown sofa, a bar with bottles, a wall decor with rockets and space-the
A woman with long dark hair lying in a hospital bed, connected to medical equipment, smiling.
A young child with glasses lying in a hospital bed, wearing a hospital gown with colorful cartoon characters, covered with white hospital sheets, and wearing a head bandage.
A young girl in a wheelchair is smiling outdoors. She has blonde hair, wears glasses, a black headband, colorful bracelets, and a pink dress with a pattern. Behind her, there is a school or community building with a brick exterior, and other children
A young girl with hospital gown lies in a hospital bed, connected to medical equipment and monitors, with a colorful rainbow coil toy nearby.
A young girl with blonde hair and blue eyes lying on a hospital bed with a colorful blanket and a purple shirt. She is smiling and looking at the camera.
A young girl lying in a hospital bed with her eyes closed, wearing glasses, and oxygen tubes, with medical equipment around her and photographs of children and a woman on the wall behind her.
A young girl in pajamas and glasses sitting in a chair, with a gift wrapped in brown paper and ribbon in front of her. A young boy in a red shirt and shorts, also holding a wrapped gift, smiles at the camera. They are inside a home with wooden floors
A young person in a wheelchair wearing a pink blanket with Mickey Mouse design, sitting in a room with a white closet door decorated with butterfly stickers and a colorful hanging toy or decoration in the background.
A young girl with glasses in a pink and black wheelchair, being attended to by a nurse or caregiver who is gently touching her forehead. The background shows a cluttered room with shelves, drawers, and various medical or craft supplies. The girl appe
A young child with blond hair lying in a hospital bed, wearing an oxygen mask, and smiling. The child is dressed in a purple pajama with a skeleton pattern. The bed is covered with a white sheet, and there are soft toys near the child.
A young child lying in a hospital bed, wearing a gray shirt and a pink hospital wristband. The child has a nasal cannula for oxygen and a sticker on their face. Towels and blankets are on the bed, and there is medical equipment and a bed railing in t
A young boy with closed eyes sitting in a wheelchair, looking tired or sleepy, with a Disney Toy Story themed poster in the background.
A young boy with a shaved head and a red mark on his forehead, wearing an orange T-shirt with blue trim. He appears to be sitting and looking to the side, with a slightly concerned or curious expression.
A young child with curly hair and glasses, smiling at the camera, wearing a bib with text, in a kitchen setting.
A young boy with dark hair and dark eyes sitting in a black and blue wheelchair, looking up at the camera with a curious expression. He is wearing a light blue T-shirt and striped shorts, with bare feet. The background shows a tiled floor, wooden fur
Young man with short dark hair and glasses, sitting in a wheelchair, wearing a blue and black plaid shirt, smiling, in a room with chairs and some people in the background.
A young boy with short dark hair sleeping in a hospital bed, wearing an oxygen mask attached to a tube, with a blue blanket and colorful pajamas.
Two children with disabilities in wheelchairs sit in front of a decorated Christmas tree with ornaments and lights. The girl on the left has dark curly hair, is smiling, and wears a pink dress with gold accents. The boy on the right has short dark ha
A young girl lying in a hospital bed with medical tubes attached to her chest, smiling with her eyes closed, wearing pink pajamas with colorful animal and nature prints. She has a decorative bow on her head and is resting her head on a pink pillow wi
A young girl sitting on a man's lap with a woman next to them, all smiling at the camera inside a house.
A young girl in a wheelchair with a pink pillow and blanket, wearing white shoes and light-colored pants. She appears to be resting or lost in thought. The wheelchair is black and pink, and the floor is carpeted with a beige pattern.
A young girl in a wheelchair looking at the camera inside a living room.
Young child lying in a hospital bed with medical equipment and wires attached, covered with a pink floral blanket. The scene appears to be in a hospital setting.
Close-up of a newborn baby wrapped in a colorful polka dot blanket lying in a hospital crib with medical equipment nearby.
A young boy with dark hair lying in bed, wearing a dark gray shirt and sweatpants. He has Spider-Man themed pillowcases and sheets, and a bright red pillow behind his head. The boy is wearing red and black Spider-Man socks, and there are some wires o
A young girl with glasses and a medical tube lying in bed, holding a Minnie Mouse stuffed toy, surrounded by colorful heart graphics.
A sign encouraging donations to fund treatment for Frizzle patients, with text in various shades of blue and green and a heart icon.
DONATE TODAY

It wasn't always like this for children with FRRS1L gene disorder.

Before the disorder took full effect, parents knew and saw their children to be busy toddlers. FRRS1L is a critical brain encoding gene that affects all cells in the brain. Children with this gene disorder develop, albeit delayed, until age two when they begin having seizural activity that causes regression and total loss of function.

Learn more about FRRS1L

REGISTER HERE
A digital graphic promoting registration for children with newly diagnosed Frizzle (FRRS1L). It features a white background with teal and blue accents, a heart logo with DNA strands, and icons of a clipboard with a check mark and a family symbol with a heart.