THE CAUSE
We’re raising a voice for all kiddos with Zellweger Spectrum Disorder.
Zellweger Spectrum Disorder is a rare, genetic disorder that is usually terminal in childhood.
The disorder affects every system in a person’s body. In Zellweger Spectrum Disorder the peroxisomes in the body’s cells aren’t working properly, are absent, or are severely decreased. Peroxisomes are present in almost every cell of the body, so most bodily functions are affected.
Patients with Zellweger may experience deafness, blindness, developmental delays, adrenal insufficiency, neurological issues, and feeding issues.
The difficult part of this journey is that the disorder manifests itself differently in each individual. Every child has a different experience, different challenges, differing qualities of life, and a unique story.
Currently, there are no effective cures for peroxisomal disorders. Instead, management and treatment of symptoms are crucial to help patients with peroxisomal disorders live longer, healthier lives.
3,000
babies are born annually with a peroxisomal disorder
95%
of rare diseases do not have any FDA approved treatment - including peroxisomal disorders
1 in 10
people are diagnosed with a rare disorder. That’s more than cancer and AIDS combined
The Research Your Support Makes Possible
Hope With Lo exists to accelerate meaningful progress for children living with Zellweger Spectrum Disorder.
In partnership with the Global Foundation for Peroxisomal Disorders, we are helping advance Project PEX1-VISION - a new treatment designed to preserve vision in children with Zellweger Spectrum Disorder.
Unlike many research initiatives that are still in the discovery phase, Project PEX1-VISION has already reached several significant milestones:
Comprehensive preclinical proof-of-concept completed
Intellectual property secured
Clinical-grade manufacturing underway
Regulatory engagement initiated
Preparing for first-in-human clinical trials
Researchers have demonstrated encouraging preclinical results, including improved vision, preservation of retinal cells, prevention of retinal degeneration, and sustained benefit after a single treatment in animal models.
Today, the greatest need is funding the final safety studies required before human clinical trials can begin.
When you register, donate, sponsor Hope With Lo, or become a fundraising team, you're helping accelerate this work.
Meet other Zellweger Warriors
Jaley - 3.09.18 - 3.28.23 Jaley loved riding around her her convertible Mercedes and she loved Annie's cheddar bunnies, but only the cheddar and they had to be whole or she would mic drop them.
Ruby (Roo) - 6.17.17 - 6.22.22 Her laugh and personality were infectious. She had a glowing smile, stunning eyes and an undeniable calmness. Her love of chips was only rivaled by her love of dancing. She mastered the lasso high above her head with her favorite rubber noodles. She loved a rough ride in her car or wheelchair and she even enjoyed the few times she fell out of the thing. She was a beautiful, one-of-a-kind kiddo that will never be forgotten.
Camila - 9.13.21 - 3.27.23 Camila loved to take baths and feel the water on her face. She loved being held by her mom and laying on her chest.
Lucy - 9.10.18 Lucy is full of life with an infectious laugh. She finds fun in everything and is always up to mischief. She loves listening to music, Moana and Frozen.
James (J.W.) 4.23.04 - 4.20.05 J.W.'s favorite thing was looking at lights and being tickled. He loved to scream and laugh as he swung his arms and kicked his legs...we said he was playing with his angels.
Ema - 6.29.22 - 4.08.23 Ema loved drinking her milk so much so that when she moved to a G-tube, she would still move her lips as if she were drinking it. She used to calm down and fall asleep to 'Frère Jacques'.
Saint - 6.03.22 - 9.10.22 Saint's favorite thing was hearing mama's voice or being in his mama's arms. Every time he was held, his oxygen and heart rate would jump from excitement. He made it known that he loved his mama most!
Austin - 6.22.12 - 11.08.14 Austin was so silly and his smile lit up the room. He loved his swing and to be held by mama.
Brylee Grace - 8.27.20 Brylee loves her daddy's beard, her sisters being rough with her, putting anything that vibrates in her ears, and playing with hair. She likes to get loud and laugh when everyone is quiet - she recently got tickled at a funeral during a very quiet time, LOL!
Patrick - 8.08.2013 Patrick's nickname started as bean went to bug and now is bubba. Patrick loves Mickey Mouse and Cat in the Hat. He loves riding on the golf cart and going camping. My favorite memory is when he took his first steps. We waited 2 1/2 years and then it happened like magic!
Research means hope.
There are new breakthroughs happening in medicine daily.
Research is being done and promising work in gene therapies have made headway in other leukodystrophies. Someday it will happen for Zellweger, too.
Why not now?
Why not be the ones to help usher in what will one day change the life of a kiddo like Lo? Research will lead to breakthroughs for Lo but also balloon into hundred of thousands of people who can benefit.
100% of donations go to research for new treatments.
100% of everything we raise will be donated to the Global Foundation of Peroxisomal Disorders and will be directly applied to fund research exclusively dedicated to Zellweger Spectrum Disorder.
