Overview
Morquio Syndrome is treated with 2 medications in our database, including VIMIZIM, ELAPRASE. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by BioMarin, Takeda. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Morquio 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 eventsRadicle Science — NA
University of Nottingham — NA
University Hospital, Montpellier — NA
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableVIMIZIM
* 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
ELAPRASE
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
Clinical Trials
View all trials with filters →No actively recruiting trials found for Morquio Syndrome at this time.
New trials open frequently. Follow this disease to get notified.
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 Morquio Syndrome.
Community
No community posts yet. Be the first to share your experience with Morquio Syndrome.
Start the conversation →Latest news about Morquio Syndrome
Disease timeline:
New recruiting trial: Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation
A new clinical trial is recruiting patients for Morquio Syndrome
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 Morquio Syndrome
What is Morquio Syndrome?
Morquio Syndrome is treated with 2 medications in our database, including VIMIZIM, ELAPRASE. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by BioMarin, Takeda. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Morquio Syndrome treatment below.
How is Morquio Syndrome inherited?
Morquio Syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Morquio Syndrome typically begin?
Typical onset of Morquio Syndrome is childhood. Age of onset can vary across affected individuals.
Which specialists treat Morquio Syndrome?
5 specialists and care centers treating Morquio 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 Morquio Syndrome?
2 FDA-approved treatments and 1 patient support program are currently tracked on UniteRare for Morquio Syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.