OUR KIDS ARE DYING! PLEASE HELP BY FUNDING THIS CAMPAIGN TO GET OUR GENE THERAPY UP AND RUNNING TO SAVE OUR CHILDREN'S LIVES!
The Sebastian Velona Foundation, in partnership with Caleb's Chance Foundation and Cowboy Brock's Cause For A Cure Foundation are asking for your help in funding a gene therapy trial that will potentially save our children's lives, and the lives of all children impacted by Batten Disease CLN8.
Sebastian's Journey: In October of 2007, Sebastian Velona collapsed with his first seizure -- he was hospitalized and then told to see his doctor. Ultimately, Sebastian was diagnosed with epilepsy and, until doctors found the right medicine adn dosage, Sebastian's seizures grew more sever and frequent. Within the next year, we noticed some behavioral changes in Sebastian, but we thought it had to do more with the medication and the hardship of having seizures. At around 6 years old, we noticed that Sebastian was having difficulty reading, began slurring his speech, falling behind in school, stumbling while walking, and needing more and more help with even the most routine daily activities. Ultimately, Sebastian underwent DNA testing at UCLA and was diagnosed with Batten CLN8. For several years we searched for any and all treatments but there were non.
Caleb's Journey: Around two and a half years old, Caleb Johnson's language seemed to plateau, and he was showing some fine motor delays. He became so impulsive it was difficult to keep him on task. Caleb was then enrolled in preschool in hopes that he would catch up, but as time passed Caleb became more, and more fixated on his favorite movies, and objects. Suddenly, Caleb wasn’t answering, no matter who called his name. Our suspicions were confirmed when Caleb was diagnosed with autism after his third birthday.In September of 2017, Caleb was only a few months from his 6th birthday. Our family, and his community were all celebrating more than two months seizure free for Caleb. He seemed to be more alert, and more excited than recent months. He was enjoying socializing, and engaging in school activities. We were all hoping to see him regain a lot of his lost skills until we finally received a diagnosis. Genetic testing proved that Caleb had all along been suffering from late infantile variant batten disease. Suddenly, we were just wishing we didn’t have the answers we had been so desperately seeking. We found that Battens disease is an extremely rare neuro-degenerative disease that devastates children and their families. Children with Caleb’s type of batten are certain to lose their ability to see, walk, talk, and are left bedridden before an early death between 8 and 12 years old. We were told there are currently no treatments, and no cure available for batten.
Brock's Journey: In October of 2013, Brock Scarpetta's teacher noticed that one day he would say and recognize the alphabet and numbers and the next day he couldn’t do it. At home, we noticed he was falling more often than a boy his age should be. We found a tutor to help Brock at home. It seemed to help a little. We thought the more repetition the better. Then I took him to see a psychologist. He diagnosed Brock with Auditory Processing Disorder. Then on May 1, 2014 Brock had his first grand-mal seizure. After hours of being there, the doctors determined that this was a febrile seizure. June 26th Brock had not one but two grand-mal seizures back to back. Again, we ended up at the emergency room where they admitted Brock. He had an MRI and blood work taken. They diagnosed him with Epilepsy July 2014. We were then referred to UW Children’s Hospital in Madison, WI. October of 2014, doctors at UW did a panel of blood work to see if they could determine a diagnosis for Brock. All tests came back negative. The genetic doctor then suggested we do a skin biopsy. It was a good six months of getting this type of testing approved through insurance. Meanwhile, Brock suffered another seizure. It was then June 2015, Brock had the skin biopsy done. July 2015, we received the results. Brock tested positive and was diagnosed with Batten Disease CLN8 variant. The doctor said this was a rare and fatal autosomal disease with no cure or treatment.
How you can help: Through your continued support, we are able to ensure research will be used to hopefully find a cure and that no other families have to go down the road of misdiagnosis until they are eventually told the words no parent ever wants to hear: “There is no cure.”. It is our goal to expand research so as to help all children suffering from Batten’s Disease and provide support to all families, regardless of what stage they are in, to ensure they have the necessary tools and support systems to triumph during these difficult times. Our immediate needs are to raise close to $1,000,000 for research and a clinical trial. We are at 35% to goal to date and are hoping to be at 100% within twelve months. The above listed foundations have partnered with The Giving Back Fund, a national nonprofit specialising in philanthropic management and fiscal sponsorship. This structure ensures your donation goes to programming and not overhead.
What is Batten's Disease: Batten disease is a rare, genetically inherited disorder, which belongs to a group of progressive degenerative neurometabolic disorders, known as the Neuronal Ceroid Lipofuscinoses (NCLs). Neuronal Ceroid Lipofuscinoses (NCLs) are characterized by genetic mutations which disrupt cells’ ability to dispose of wastes, resulting in the abnormal accumulation of certain proteins and lipids (fats) within the nerve cells of the brain and other tissues of the body, resulting in progressive neurological impairment including developmental regression, seizures, blindness, behavior changes and dementia. There are many forms of neuronal ceroid lipofuscinoses. Mutations in at least eight different genes are known to cause Batten disease.
More information can be found by going to any of the foundation's websites here:
https://www.curebattencln8.org/
https://www.breakbatten.org/
https://www.cowboyforacause.com/
