![\"Connor](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2026\/07\/SUT-L-CONNOR-002.jpg\")
Connor Dalby, 17, and his mom Kelley Del Real take a walk together in the hallway at Rady Children’s Hospital. (Meg McLaughlin \/ The San Diego Union-Tribune)<\/p>\nConnor Dalby could not walk for the first 14 years of his life, so his newfound ability to scamper down hallways is miraculous on its own.<\/p>\n
But the experimental drug that reduced his severe and frequent seizures by 90% has also conveyed a host of other benefits. For his mother, Kelley Del Real, other improvements are equally transformative, not just for Connor, but for a family that has cared for him around the clock since his symptoms appeared a few days after his birth.<\/p>\n
\u201cYes, the walking was a big surprise, but he has also gained the ability to use his hands for different things that he wasn\u2019t able to do before,\u201d Del Real said. \u201cHe is a lot calmer overall.<\/p>\n
\u201cBefore, he was constantly irritable and just always uncomfortable, but that has shifted a lot. It has improved his sleep, and, overall, it\u2019s a huge change in our quality of life.\u201d<\/p>\n
No, this drug has not been a universal fix.<\/p>\n
Connor Dalby, 17, and his mom Kelley Del Real take a walk together in the hallway at Rady Children’s Hospital. (Meg McLaughlin \/ The San Diego Union-Tribune)<\/p>\n
Connor, 17, is still on the severe end of the autism spectrum with developmental and epileptic encephalopathy, a rare form of epilepsy. He still cannot speak for himself and, while he is able to stand on his own and excitedly move where he wants to go, he still often needs guidance from a caregiver.<\/p>\n
And the drug, called an \u201cantisense oligonucleotide,\u201d or \u201cASO,\u201d is far less fast-acting than, say, a pair of aspirin to treat a stubborn headache.<\/p>\n
\u201cThe effects wear off a week or two before his next dose, and then the effect of the dosing isn\u2019t immediate; it takes another week or two to take effect,\u201d Del Real said.<\/p>\n
The lag between one dose, delivered directly into his spinal cord, and the next allows some symptoms to return.<\/p>\n
But the payoff is well worth the effort.<\/p>\n
\u201cWe get a solid five, six weeks where he\u2019s doing really well with everything,\u201d Del Real said.<\/p>\n
And, some benefits do persist even when the drug has worn off or not yet taken effect. Previously, Connor was in the habit of screaming every time a vehicle he was riding in slowed down. That tendency has gone away entirely. And his habit of waking up for hours at 2 or 3 a.m. every night has also ended.<\/p>\n
\u201cNow, he goes to sleep at about 9:30 at night, and he sleeps until about 4:30 a.m., which, for him, that\u2019s phenomenal,\u201d Del Real said. \u201cWe\u2019re all able to sleep.\u201d<\/p>\n
While her son still needs round-the-clock care, the situation is much less dire than it was at age 2 when seizures were so frequent that Connor had to be temporarily placed on hospice status.<\/p>\n
\u201cIt was clusters of seizures all day, all night,\u201d Del Real said. \u201cAfter 50 or 100, you know, you\u2019d just lose count.\u201d<\/p>\n
Those days were the hardest, because the seizures seemed to lock Connor in developmental stasis.<\/p>\n
\u201cHe did nothing but sort of lie on his back on a feeding tube,\u201d she said. \u201cHe didn\u2019t smile anymore, there was no laugh, I certainly didn\u2019t think that there was anything going on with him.<\/p>\n
\u201cLike, I didn\u2019t think he knew I was his mother.\u201d<\/p>\n
By age 4, there was still no precise explanation for Connor\u2019s seizures, and he was buried under the overlapping side effects of multiple ineffective medications.<\/p>\n
![\"Kelley](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2026\/07\/SUT-L-CONNOR-007.jpg\")
Kelley Del Real shows off her tattoo as she wheels her son to the elevator at Rady Children’s Hospital on Wednesday, July 22, 2026 in San Diego, California. (Meg McLaughlin \/ The San Diego Union-Tribune)<\/p>\n
Del Real, also raising Connor\u2019s brothers Chase and Cameron, was thankful for her parents moving across the street from the family home to provide some respite. She began to experiment with diet and started removing some seizure medications that weren\u2019t working. Once he was stable enough to exit hospice, a genetic analysis spotted a random mutation in one of his SCN2A genes that caused his seizures. But there was no treatment available.<\/p>\n
Previously a high school English teacher, Connor\u2019s mom had zero experience with genetics but felt, now that modern genetic sequencing had pinpointed a cause, science ought to be able to deliver a solution. Online research indicated that seizures associated with this particular gene were often caused by malfunctioning sodium or other ion channels, the structures in neurons that provide the charge for the brain\u2019s intricate patterns of electrical activity.<\/p>\n
She learned that a few researchers were exploring certain medications that could influence the effects of SCN2A mutations and made contact with scientists and other parents on the same quest.<\/p>\n
Eventually, they started RogCon Biosciences, which licensed its therapeutic agent targeting \u201cgain of function\u201d SCN2A treatment to Praxis Precision Medicines, where Del Real now works. That drug is now in phase three clinical trials and shows promise in significantly decreasing seizures.<\/p>\n
It turned out that Connor\u2019s particular mutation did not fit the niche of the therapy.<\/p>\n
But Del Real ended up connecting with Stan Crooke<\/a>, the founder and former chief executive officer of Carlsbad\u2019s Ionis Pharmaceuticals. Crooke, she said, was stepping down to start the n-Lorem Foundation dedicated to promoting \u201cn-of-1\u201d clinical trials to help single patients with rare genetic conditions.<\/p>\n It was through this route that Connor was put in touch with Dr. Olivia Kim-McManus, who obtained special permission from the U.S. Food and Drug Administration\u2019s Investigational New Drug program, which allows administration of experimental treatments to very few patients. In this case, Connor and a patient in Chicago had medications tailored to their specific genetic characteristics and were treated under protocols reviewed and approved by the FDA.<\/p>\n Kim-McManus, a child neurologist and director of Rady\u2019s Precision Therapeutics Neuro-Interventional Program, worked with colleagues at UC San Diego and Ionis Pharmaceuticals in Carlsbad to design an ASO treatment that capitalizes on the fundamental fact that children get half their genes from their mothers and the other half from their fathers. They published<\/a> the results of this work online in the journal Nature Medicine last week, about two and a half years after Connor\u2019s first dose.<\/p>\n One of Connor\u2019s two SCN2A genes causes his brain cells to overcharge, causing seizures. But his second copy works just fine. His experimental drug knocks down signals from the mutated gene but spares those from the good copy.<\/p>\n And it appears that these benign differences in the SCN2A gene, while they do vary from person to person, do not have infinite variability. So, this ability to block a bad signal and let a good one through could generalize to patients with a wide range of harmful mutations rather than requiring scientists to make a one-off drug for each patient.<\/p>\n Detecting such mutations shortly after birth and administering a drug quickly has the potential to help children avoid the developmental delays caused by constant seizures.<\/p>\n \u201cWe have already identified numbers of patients that could potentially benefit from sequencing infants,\u201d Kim-McManus said. \u201cThere\u2019s a massive potential impact here.\u201d<\/p>\n Dr. Timothy Yu, a neurologist at Boston Children\u2019s Hospital and an assistant professor at Harvard University, recently published<\/a> trial results using ASOs to treat seizures in a pair of 2-year-old girls by more broadly targeting messenger RNA that was causing seizures.<\/p>\n![\"Dr.](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2026\/07\/SUT-L-CONNOR-005.jpg\")
Dr. Olivia Kim-McManus at Rady Children’s Hospital on Wednesday, July 22, 2026. (Meg McLaughlin \/ The San Diego Union-Tribune)<\/p>\n![\"Kelley](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2026\/07\/SUT-L-CONNOR-004.jpg\")
Kelley Del Real hugs Dr. Olivia Kim-McManus at Rady Children’s Hospital. (Meg McLaughlin \/ The San Diego Union-Tribune)<\/p>\n