Oceanside mother Kelley Dalby said she has never lost hope despite the hardships she and her son, Connor, have faced in the last 12 years. At just 8 months old, Connor was diagnosed with a form of epilepsy.
At the age of 4, Connor was diagnosed with a SCN2A mutation that caused developmental impairments. As a mother, Dalby searched for every possible treatment to help her son, but it wasn’t until she discovered the n-Lorem Foundation that she found renewed hope.
The n-Lorem Foundation, founded by San Diego biotech leader Stanley Crooke, uses antisense oligonucleotides (ASO) technology to create safe treatments for patients with “ultra rare diseases.” Crooke, who developed the technology, said he hopes to treat as many people as possible.
“I don’t feel a foundation could support us in a bigger way then n-Lorem is currently doing,” Dalby said. “It is the most innovative mission I have ever heard of or could have even imagined.”
Connor is just one of four patients who Crooke expects to provide treatment to in 2021. Next year, he expects the number to increase to 20.
He said the technology is possible because of “30 years of innovative science and billions of dollars invested.”
“Today this technology is the only technology that can be used to meet the needs of many ultra-rare patients and the only approach for which the FDA has issued guidance, but we hope that over time other technologies will mature sufficiently to bring benefit to these desperate patients,” said Crooke, who founded Carlsbad’s Ionis Pharmaceuticals.
Although the foundation has plans to treat 20 patients next year, Crooke said they’ve received more than 80 applicants from people desperately seeking support.
“We have a goal to scale, and we must scale to meet the incredible needs of the ultra-rare community so no one is denied treatment when ASO could potentially help,” he said. “It would break my heart to tell a patient we can’t help because we don’t have the funds.”
Crooke credits his circle of donors and partners who have supported the foundation, but hopes to increase his network so that he can continue to help patients for free.
In the meantime, Dalby of Oceanside is just grateful to have found a treatment plan for her son.
“What they are doing is nothing short of spectacular and it is altruism at it’s finest,” Dalby said. “I am forever grateful for this opportunity. The idea of n-Lorem has carried me through so many difficult days, knowing our future has the potential to be very different then our present.”