Overview
Batten Disease is treated with 1 medication in our database, including Brineura. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by BioMarin. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Batten Disease treatment below.
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
8 eventsNeurogene Inc. — PHASE1, PHASE2
Benjamin Greenberg — PHASE1
Northwell Health — NA
Emily de los Reyes
Jessica Scherr
Alcyone Therapeutics, Inc — PHASE1, PHASE2
Emily de los Reyes
University of Rochester
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableBrineura
* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
1 grantsBioMarin Patient Assistance Program (Brineura)
Eligibility is generally limited to individuals diagnosed with a rare disease, genetic disorder, or specific chronic condition and their caregivers. Requirements typically include a demonstrated financial need or a necessity to travel long distances for specialized medical treatment, clinical trials, or follow-up care.
Community
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1 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Batten Disease
What is Batten Disease?
Batten Disease is treated with 1 medication in our database, including Brineura. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by BioMarin. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Batten Disease treatment below.
How is Batten Disease inherited?
Batten Disease follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Batten Disease?
Yes — 6 recruiting clinical trials are currently listed for Batten Disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Batten Disease?
19 specialists and care centers treating Batten Disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Batten Disease?
1 FDA-approved treatment are currently tracked on UniteRare for Batten Disease. See the treatments and support programs sections for copay assistance, eligibility, and contact details.