Hurler syndrome

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:93473OMIM:607014E76.0
Who is this for?
Show terms as
1FDA treatments1Active trials18Specialists8Treatment centers2Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Hurler Syndrome is treated with 1 medication in our database, including ALDURAZYME. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Sanofi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hurler Syndrome treatment below.

Also known as:

Clinical phenotype terms— hover any for plain English:

Abnormal diaphysis morphologyHP:0000940
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Dec 2023A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Orchard Therapeutics — PHASE3

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

ALDURAZYME

LARONIDASE· Genzyme Corporation■ Boxed WarningOrphan Drug
Patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form who have moderate to severe symptoms. Reduces urinary glycosaminoglycans and improves

Patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form who have moderate to severe symptoms. Reduces urinary glycosaminoglycans and improves walking capacity and pulmonary function.

Clinical Trials

1 recruitingView all trials with filters →
Phase 31 trial
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
Phase 3
Active
· Sites: Minneapolis, Minnesota; Milan +3 more

Specialists

18 foundView all specialists →
PO
Paul Orchard
MINNEAPOLIS, MN
Specialist
PI on 1 active trial
PM
Paul Orchard, MD
MINNEAPOLIS, MN
Specialist
PI on 15 active trials
TM
Tippi C MacKenzie, MD
San Francisco, California
Specialist

Rare Disease Specialist

PI on 2 active trials
PM
Patricia I Dickson, MD
Specialist
PI on 3 active trials
JM
Joanne Kurtzberg, MD
DURHAM, NC
Specialist
PI on 11 active trials
SO
Shigetoyo Oguri
Specialist
PI on 1 active trial
PP
Patrice Rioux, MD PhD
Specialist
PI on 3 active trials
MP
Michael Potegal, PhD
MINNEAPOLIS, MN
Specialist
PI on 1 active trial
SM
Suhag Parikh, MD
CHICAGO, IL
Specialist
PI on 1 active trial
BM
Biljana Horn, MD
SAN FRANCISCO, CA
Specialist
PI on 2 active trials
CP
Chester B. Whitley, M.D., Ph.D.
LAS VEGAS, NV
Specialist
PI on 1 active trial
AM
Agnes Chen, MD
TORRANCE, CA
Specialist
PI on 1 active trial
PP
Patrice P Rioux, MD PhD
Specialist
PI on 2 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources
ALDURAZYME(LARONIDASE)Genzyme Corporation

1 travel grant are also available for Hurler syndrome patients — see Travel Grants below ↓

Travel Grants

1 grants

Aldurazyme Co-Pay Assistance Program

Apply ↗

Community

Open Hurler syndromeForum →

No community posts yet. Be the first to share your experience with Hurler syndrome.

Start the conversation →

Latest news about Hurler syndrome

No recent news articles for Hurler syndrome.

Follow this condition to be notified when news becomes available.

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 Hurler syndrome

What is Hurler syndrome?

Hurler Syndrome is treated with 1 medication in our database, including ALDURAZYME. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Sanofi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hurler Syndrome treatment below.

Are there clinical trials for Hurler syndrome?

Yes — 1 recruiting clinical trial is currently listed for Hurler syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Hurler syndrome?

18 specialists and care centers treating Hurler 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 Hurler syndrome?

1 FDA-approved treatment and 1 patient support program are currently tracked on UniteRare for Hurler syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.