9 California Hospitals and 12 Specialists Treating KCNT1 Epilepsy (Plus How Nome Delivers Personalized ASO Therapy)
KCNT1 mutations cause severe, treatment-resistant epilepsy with conventional medications helping only 26.7% of patients. California hosts 9 Level 4 accredited epilepsy centers and leading specialists with genetic epilepsy expertise. More importantly, Nome provides personalized antisense oligonucleotide (ASO) therapy targeting your child's specific KCNT1 mutation rather than just managing symptoms. This guide lists California's top hospitals, specialists, and how Nome's 5-step process moves families from diagnosis to personalized treatment with transparent pricing and expert coordination.
What You Need to Know About KCNT1 Before Reading This List
KCNT1 mutations increase potassium channel activity up to 22-fold above normal, causing:
Epilepsy of Infancy with Migrating Focal Seizures (EIMFS): Seizures start in first 6 months, mortality 17-33%, KCNT1 causes 50% of all EIMFS cases
Autosomal Dominant Nocturnal Frontal Lobe Epilepsy (ADNFLE): Seizures during sleep, mild-to-moderate intellectual disability
Birth incidence: 1.1 per 100,000 live births (approximately 3,000 cases worldwide)
Current treatment success: Only 26.7% respond to standard medications
Best non-drug option: Ketogenic diet shows 43.5% response rate
Diagnosis requires genetic testing via whole exome sequencing or epilepsy gene panels. No FDA-approved therapies exist specifically for KCNT1, which is why Nome's personalized approach matters.
9 California Level 4 Epilepsy Centers for KCNT1 Treatment
1. UCSF Benioff Children's Hospitals (San Francisco & Oakland)
Why it's on this list: 10 dedicated epilepsy monitoring rooms at Parnassus, 5 at Mission Bay
What they offer:
24/7 video-EEG monitoring
SEEG, laser ablation (LITT), responsive neural stimulation (RNS)
Active participation in KCNT1 research and ASO therapy clinical trials
Dr. Joseph E. Sullivan directs pediatric epilepsy program
Contact: (415) 353-2437 | epiladminstaff@ucsf.edu | UCSF Epilepsy Program
2. Lucile Packard Children's Hospital at Stanford (Palo Alto)
Why it's on this list: Dedicated Pediatric Epilepsy Genetics Clinic for ion channelopathies like KCNT1
What they offer:
ROSA robotized surgical assistant (first in Northern California)
Genetic counseling through Pediatric Neurogenomics Program
Dr. Brenda Porter directs pediatric epilepsy
Dr. Juliet Knowles runs California Synaptopathy Clinic for genetic epilepsies
Contact: (844) 868-4345 | Stanford Epilepsy Center
3. UC Davis Health (Sacramento)
Why it's on this list: 2025 funding from Hartwell Foundation and KCNT1 Epilepsy Foundation for active KCNT1 research
What they offer:
Dr. Sarah Olguin and Dr. Jill Silverman developing rodent models of KCNT1 epilepsy
Level 4 accreditation for pediatric and adult services
Research leadership in KCNT1 therapeutics
Contact: (916) 734-2011 | UC Davis Epilepsy Program
4. Children's Hospital Los Angeles (CHLA)
Why it's on this list: Only Level 4 center in a free-standing children's hospital in Los Angeles with on-site genetic testing
What they offer:
Center for Personalized Medicine provides rapid genetic identification for clinical trial enrollment
Over 75 epilepsy surgeries per year with highest seizure-free rates
New-Onset Seizure Clinic: 7-10 day access after first seizure
Dr. Sucheta Joshi serves as Medical Director
Epilepsy Neurogenetics Fellowship Program (one of only 5 in the United States)
Contact: (323) 361-2471 | CHLA Epilepsy Center
5. Rady Children's Hospital San Diego
Why it's on this list: #8 nationally in pediatric neurology, Dr. Kim-McManus conducts KCNT1 drug trials
What they offer:
Institute for Genomic Medicine with advanced genomic sequencing
Rady Precision Medicine Clinic
Dr. Olivia Kim-McManus treats KCNT1 patients and runs industry-sponsored trials
ROSA robotic assistance for precision procedures
Contact: (858) 966-5819 | Rady Epilepsy Program
6. CHOC (Children's Hospital of Orange County)
Why it's on this list: California's first children's hospital named Level 4 center, only Level 4 in Orange County
What they offer:
Dr. Maija-Riikka Steenari treats KCNT1 patients specifically
Dravet Syndrome Center of Excellence
Two epilepsy monitoring units (Orange and Mission Viejo)
1,200+ patients monitored yearly
Contact: (888) 770-2462 | CHOC Epilepsy Program
7. UCLA Mattel Children's Hospital (Los Angeles)
Why it's on this list: NORD-designated Center of Excellence, pioneered PET scans for childhood epilepsy
What they offer:
California Center for Rare Diseases
Dr. Lekha M. Rao directs Pediatric Epilepsy Fellowship
Ketogenic Diet Program (keto@mednet.ucla.edu) showing 43.5% KCNT1 response rates
Triple board-certified epilepsy specialists
Contact: (310) 825-0867 | childneuro@mednet.ucla.edu | UCLA Pediatric Neurology
8. USC Keck Medicine Comprehensive Epilepsy Center (Los Angeles)
Why it's on this list: Largest surgical epilepsy program on West Coast, hub of USC Epilepsy Care Consortium
What they offer:
Network linking 6 epilepsy centers across Central and Southern California
Equitable access for underserved communities
Connection to CHLA, Rancho Los Amigos, Hoag Hospital
Contact: (800) 872-2273 | USC Epilepsy Center
9. Loma Linda University Children's Hospital (Inland Empire)
Why it's on this list: Serves Riverside and San Bernardino regions, First Seizure Clinic with 2-week access
What they offer:
Level 4 pediatric epilepsy center
First responsive nerve stimulator (RNS) program in Inland Empire
CCS clinic for medically intractable epilepsy
Fastest response time in California for first seizure patients
Contact: (909) 558-4000 | Loma Linda Pediatric Epilepsy
12 Leading California KCNT1 Specialists You Should Know
KCNT1 Foundation-Listed Physicians (Direct KCNT1 Experience)
1. Dr. Olivia Kim-McManus, MD (Rady Children's Hospital - San Diego)
Credentials: Board certified Neurology, Child Neurology, Epilepsy
Why she's on this list:
Actively conducts industry-sponsored KCNT1 drug trials
Director of Pediatric Epilepsy Fellowship Program at UC San Diego
Specializes in medically intractable epilepsy requiring surgery, laser ablation, neuromodulation devices
Contact: (858) 966-5819 | 3020 Children's Way, San Diego | 2125 Citracado Parkway, Escondido
2. Dr. Brenda E. Porter, MD, PhD (Stanford University)
Credentials: Professor of Neurology, Director of Pediatric Epilepsy
Why she's on this list:
Listed on KCNT1 Foundation physician directory
Leads multicenter trial for biomarker availability in rare genetic epilepsy
Co-directs Tuberous Sclerosis Clinic
Develops therapies for epilepsy prevention in genetic epilepsies
Trains future leaders through NIH Child Neurologist Career Development Program
Contact: (650) 723-0993 | brenda2@stanford.edu
3. Dr. Maija-Riikka Steenari, MD (CHOC - Orange County)
Credentials: Neurology specialist
Why she's on this list:
KCNT1 Foundation-listed physician
Treats KCNT1-related disorders directly
Practices at Orange County's only Level 4 pediatric epilepsy center
Contact: (888) 483-5670 | (714) 509-7601 | 201 W La Veta Ave, Orange
Stanford University Genetic Epilepsy Specialists
4. Dr. Juliet K. Knowles, MD, PhD (Stanford)
Credentials: Assistant Professor Neurology, Founding Physician of California Synaptopathy Clinic
Why she's on this list:
Specialty care for SYNGAP1, STXBP1-related epilepsy and similar genetic epilepsies
2022 Nature Neuroscience publication on myelin plasticity in pediatric epilepsy
Independent laboratory studying mechanisms underlying seizures and cognitive dysfunction
Funding from American Epilepsy Society, NIH/NINDS, CURE Epilepsy Foundation
Contact: Available through Stanford Epilepsy Genetics Clinic
5. Dr. William Gallentine, DO (Stanford)
Credentials: Clinical Professor, Chief of Division of Child Neurology
Why he's on this list:
Multi-disciplinary care for genetic epilepsy syndromes with mutation-specific therapy focus
Research on inflammation and genetics in epilepsy development
11 years as pediatric epileptologist at Duke before joining Stanford 2018
Contact: Available through Stanford Child Neurology
6. Dr. Ann Hyslop, MD (Stanford)
Credentials: Clinical Associate Professor, Director of Pediatric Epilepsy Fellowship
Why she's on this list:
Conducted 15+ clinical trials in pediatric medically intractable epilepsy
Develops neuromodulation program for pediatric epilepsy at Stanford
10 years experience at Miami Children's Hospital before Stanford
Contact: Available through Stanford Epilepsy Center
7. Dr. Fiona Baumer, MD, MS (Stanford)
Credentials: Assistant Professor Neurology, MS Epidemiology and Clinical Research
Why she's on this list:
Specializes in difficult-to-treat pediatric epilepsies
Runs Stanford's clinical transcranial magnetic stimulation (TMS) program
Research on how epilepsy affects language acquisition and brain connectivity
Training at Boston Children's Hospital, Stanford's first Maggie Otto Fellow
Contact: Available through Stanford Pediatric Neurology
UCSF Benioff Specialists
8. Dr. Joseph E. Sullivan, MD (UCSF)
Credentials: Professor Neurology and Pediatrics, Director of UCSF Pediatric Epilepsy Center of Excellence
Why he's on this list:
Expertise in Dravet syndrome and genetic epilepsies
Board of Directors for Dravet Syndrome Foundation
Chairs PCDH19 Alliance scientific advisory board
Research on cognitive dysfunction and mood disorders in epilepsy
Contact: (415) 353-2437
9. Dr. Vikram Rao, MD, PhD (UCSF)
Credentials: Oversees UCSF Epilepsy Division
Why he's on this list:
Expertise spans medications, surgery, and implanted neurostimulation devices
Uses direct brain electrical recordings and implanted devices to map seizure patterns
Bridges research and clinical care
Contact: (415) 353-2437
UCLA and CHLA Leadership
10. Dr. Lekha M. Rao, MD (UCLA)
Credentials: Triple board-certified (Neurology/Child Neurology, Clinical Neurophysiology, Epilepsy)
Why she's on this list:
Director of Pediatric Epilepsy Fellowship track at UCLA
Associate Program Director for Child Neurology Residency
Los Angeles Magazine and Los Angeles Times Top Doctor
Principal investigator on clinical trials for pediatric and neonatal seizures
2011-2013 Epilepsy Foundation of Greater Los Angeles Care and Cure Fellowship
Contact: (310) 825-6196 | childneuro@mednet.ucla.edu
11. Dr. Sucheta Joshi, MD, MS, FAAP, FAES (CHLA)
Credentials: Medical Director Comprehensive Epilepsy Center, Professor Pediatric Neurology USC
Why she's on this list:
Co-directs Epilepsy Neurogenetics Fellowship Program (one of only 5 in United States)
Secretary of Board of Pediatric Epilepsy Research Consortium
Advisory Committee for AAP National Coordinating Center for Epilepsy
Led Epilepsy ECHO projects since 2013 expanding care to underserved areas
Contact: (323) 361-2471
12. Dr. Matthew Deardorff, MD, PhD (CHLA)
Credentials: Director Center for Personalized Medicine, Co-director Epilepsy Neurogenetics Fellowship
Why he's on this list:
Expertise in medical genetics and neurogenetic disorders
On-site genetic testing enables rapid KCNT1 patient identification for clinical trials
Integration of genetic testing into epilepsy diagnosis and treatment
Contact: (323) 644-8528 | askcpm@chla.usc.edu
5 California State Resources Providing Free Services for KCNT1 Families
1. California Children's Services (CCS)
What they provide:
Diagnostic and treatment services
Medical equipment
Medical case management
Physical and occupational therapy
High Risk Infant Follow-Up Program
Eligibility myth debunked: NOT limited to families earning under $40,000—higher income families may qualify if medical expenses are high
Contact: Your local county public health department | DHCS CCS Information
2. California Regional Centers (21 statewide)
What they provide (FREE regardless of income):
Eligibility assessment
Case management/service coordination
Individual Program Plan development
Early intervention (ages 0-3)
Equipment and therapy referrals
Respite care
Family support grants
Major centers:
Regional Center of Orange County: 26,000+ residents
San Diego Regional Center: San Diego and Imperial counties
Find yours: Department of Developmental Services listing
3. Epilepsy Foundation of Northern California
Coverage: 44 counties from Oregon border to Fresno County
Services:
Support groups (adults, teens, parents, seniors, Spanish speakers)
Mentoring and case management
Educational training
Seizure first aid trainings
Camps and activities
School support
Contact: Epilepsy Foundation Northern California | 1-800-332-1000 (24/7 helpline)
4. Epilepsy Foundation Los Angeles
Coverage: Los Angeles, San Bernardino, Ventura counties
Services:
Bilingual information and support
Individual advocacy assistance
Kids Crew program (ages 14 and under)
Family weekend camps
Teen retreats
Contact: 1-800-564-0445 (9 AM-5 PM M-F) | 1-800-332-1000 (24/7 English) | 1-866-748-8008 (24/7 Spanish) | Epilepsy LA
5. Epilepsy Foundation San Diego County
Coverage: San Diego and Imperial counties
Services:
Virtual monthly support groups (free with RSVP)
Annual Epilepsy Education Conference
#MyEpilepsySDStory storytelling initiative
Contact: 1-619-296-0161 | Epilepsy San Diego
3 NORD Centers of Excellence in California for Rare Disease Care
1. UCLA California Center for Rare Diseases
Director: Dr. Stanley Nelson
Opened: 2019 as part of UCLA Institute for Precision Health
What they offer:
Clinical trials
Specialized registries
Genetic testing
Innovative treatments for genetic disorders, neurodevelopmental disorders, metabolic conditions
Contact: UCLA Health Rare Diseases
2. Stanford Medicine Children's Health
Director: Dr. Natalia Gomez-Ospina
Coverage: Northern California with 65+ locations
What they offer:
Individualized care
Genetic testing
Innovative procedures
Contact: Stanford Children's Health
3. CHOC and UC Irvine Health (Joint Program)
Specialization: Only MPS program in Western United States, only West Coast Batten Disease Center of Excellence
Key physicians: Dr. Jose Abdenur (CHOC), Dr. Virginia Kimonis (UCI Health)
Contact: CHOC Metabolic Disorders
Why Current KCNT1 Treatments Fall Short
Standard approaches fail most KCNT1 families:
Medications: Only 26.7% achieve 50% seizure reduction
Levetiracetam, clobazam, valproic acid, topiramate, phenobarbital, benzodiazepines
Most patients fail 5+ medications
Ketogenic Diet: 43.5% response rate (best current option)
Available at UCLA (keto@mednet.ucla.edu), Stanford, UCSF, CHLA, Rady, CHOC
Requires medical supervision
31% of caregivers rate it most successful
Vigabatrin: 36% success rate for epileptic spasms specifically
Particularly effective for KCNT1 patients with West syndrome
Requires vision monitoring
Cannabidiol (Epidiolex): 14% rate it most successful
Pharmaceutical-grade CBD preferred over CBD oils
Quinidine: Only 20-30% achieve 50% seizure reduction
Cardiac side effects severely limit dosing
Cannot reach therapeutic brain levels due to toxicity
Not recommended as first-line approach
The problem: No FDA-approved therapies exist specifically for KCNT1. Current treatments manage symptoms without addressing the root cause—exactly what Nome's personalized ASO therapy changes.
How Nome's 5-Step Process Delivers Personalized ASO Therapy for KCNT1
Nome provides the structured pathway from diagnosis to personalized treatment with transparent pricing and expert partner coordination. Here's exactly how it works:
Step 1: Intake & Eligibility (FREE)
What happens:
Share genetic testing results and medical records
Nome's AI-powered system analyzes your child's specific KCNT1 mutation
Determines if mutation is amenable to ASO technology
Turns "intake chaos" into development-ready plan using data-driven logic
Why it matters: KCNT1 gain-of-function mutations are ideal ASO candidates—reducing expression restores normal channel activity
Cost: Free assessment, no financial commitment
Step 2: 30-Day Therapeutic Plan Development
What happens in ~30 days:
Molecule template design for your child's specific mutation
Target identification
Safety and toxicology study planning
Chemistry/manufacturing/controls (CMC) roadmap
Program economics breakdown
What you get: "What's next" roadmap showing:
Exact steps from diagnosis to treatment delivery
Realistic timelines
Expected costs
Milestone-based pricing structure
Cost: Transparent monthly fee after free intake, milestone-based pricing (not full upfront payment)
Step 3: Partner Orchestration
What Nome coordinates:
ASO design and synthesis with specialized laboratories
Preclinical testing in relevant KCNT1 models
Manufacturing to GMP standards
IND-enabling toxicology studies
Regulatory submission preparation
What you avoid: Trying to identify and contract with vendors independently
The technology: Knockdown gapmers that reduce KCNT1 mRNA through RNase H-mediated degradation (same proven technology behind FDA-approved ASO drugs)
Step 4: Approvals & Safety Oversight
What happens:
Rigorous laboratory safety testing
FDA permission via Investigational New Drug (IND) application
Single-patient IND pathway or expanded access framework
Nome guides families through FDA submission
Safety monitoring protocols established
What you get: Clear expectations and support working with your medical team
Step 5: Delivery to Patients & Ongoing Management
Treatment delivery:
Intrathecal injection (lumbar puncture into spinal fluid) every 2-4 months
Administered at your treating California hospital
ASO reaches approximately 90% of brain and spinal cord cells
Monitoring:
Seizure frequency tracking
Developmental progress assessment
Side effect monitoring
Clinical team coordination
Who manages what: You manage Nome; Nome manages vendors, execution, and coordination
Why ASO Therapy Specifically Targets KCNT1's Root Cause
The problem: KCNT1 gain-of-function mutations increase potassium channel activity up to 22-fold above normal
The solution: ASO gapmer technology knocks down KCNT1 expression, reducing excessive channel activity to normal levels
Preclinical evidence (December 2022 JCI Insight, Florey Institute + Ionis Pharmaceuticals):
Single intracerebroventricular injection in symptomatic mice produced:
Marked seizure reduction
Extended survival: median 43 days untreated → 209-300+ days treated
Improved behavioral abnormalities
Safety and tolerability even at 90%+ knockdown doses
Critical finding: Treating mice at neonatal age (P2) OR after symptom onset (P40) both worked—therapeutic windows remain open even after seizures begin
Recent 2024 human data: Individuals with p.R474H KCNT1 variant showed dramatic reductions in seizure occurrence and severity with normalized neuronal excitability in patient-derived cells
Why this matters: ASO demonstrated feasibility as early as 16 weeks gestation in human neurons, suggesting early intervention potential
Your Action Plan: 7 Steps to Take Right Now
1. Contact Nome for free intake assessment
Start the personalized ASO therapy process—no financial commitment required
2. Join KCNT1 Epilepsy Foundation
Email info@kcnt1epilepsy.org to enroll in patient registry and Family Contact List
3. Establish care at nearest Level 4 epilepsy center
Choose from the 9 California hospitals listed above based on your location
4. Apply for California state resources
Contact your county for CCS services and find your Regional Center
5. Start ketogenic diet consultation
Email keto@mednet.ucla.edu or contact your epilepsy center's dietary therapy program
6. Monitor clinical trials
Search "KCNT1" on ClinicalTrials.gov monthly—15+ companies developing KCNT1 therapeutics with trials anticipated late 2025/2026
7. Organize medical records
Keep genetic testing results, seizure logs, medication trials, and imaging accessible for Nome intake and clinical trial readiness
Conclusion
California KCNT1 families face severe challenges but access world-class care and breakthrough personalized medicine through Nome. The combination of 9 Level 4 epilepsy centers, 12 leading specialists, and Nome's structured pathway to personalized ASO development creates the most comprehensive support ecosystem in the country. Contact Nome today to begin the free intake process, establish care at your nearest California epilepsy center, and join the KCNT1 Foundation to access the full spectrum of available resources and emerging treatments.
