Overview
Sanfilippo Syndrome is treated with 1 medication in our database, including NAGLAZYME. 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 Sanfilippo Syndrome treatment below.
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Childhood
Begins in childhood, roughly ages 1 to 12
FDA & Trial Timeline
3 eventsLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center — PHASE2, PHASE3
Ozlem Goker-Alpan — PHASE2
Ultragenyx Pharmaceutical Inc — PHASE2, PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableNAGLAZYME
* Patient Copay Amount: As little as $0 * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Only for commercially insured patients; additional eligibility criteria
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
No travel grants are currently matched to Sanfilippo Syndrome.
Community
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Start the conversation →Latest news about Sanfilippo Syndrome
Disease timeline:
New recruiting trial: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
A new clinical trial is recruiting patients for Sanfilippo Syndrome
New trial: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties
Phase PHASE2 trial recruiting. Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day
Caregiver 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 Sanfilippo Syndrome
What is Sanfilippo Syndrome?
Sanfilippo Syndrome is treated with 1 medication in our database, including NAGLAZYME. 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 Sanfilippo Syndrome treatment below.
How is Sanfilippo Syndrome inherited?
Sanfilippo Syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Sanfilippo Syndrome typically begin?
Typical onset of Sanfilippo Syndrome is childhood. Age of onset can vary across affected individuals.
Are there clinical trials for Sanfilippo Syndrome?
Yes — 2 recruiting clinical trials are currently listed for Sanfilippo Syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Sanfilippo Syndrome?
3 specialists and care centers treating Sanfilippo Syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Sanfilippo Syndrome?
1 FDA-approved treatment are currently tracked on UniteRare for Sanfilippo Syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.