Late infantile CLN2 disease

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:699761OMIM:204500
Who is this for?
Show terms as
1Active trials5Specialists8Treatment centers

Where are you in your journey?

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

Overview

Also known as:

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Aug 2020Cerliponase Alfa Observational Study in the US

BioMarin Pharmaceutical — NA

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Late infantile CLN2 disease.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
N/A1 trial
Cerliponase Alfa Observational Study in the US
N/A
Active
PI: Medical Director, MD (BioMarin Pharmaceutical) · Sites: Little Rock, Arkansas; Orange, California +14 more

Specialists

5 foundView all specialists →
RM
Ronald G Crystal, MD
NEW YORK, NY
Specialist
PI on 12 active trials
RM
Ronald G. Crystal, MD
NEW YORK, NY
Specialist
PI on 4 active trials
DJ
David Jacoby
Specialist
PI on 1 active trial
RM
Robert Steiner, MD
Specialist
PI on 2 active trials
RM
Ronald Crystal, MD
NEW YORK, NY
Specialist
PI on 4 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

Travel Grants

No travel grants are currently matched to Late infantile CLN2 disease.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Late infantile CLN2 diseaseForum →

No community posts yet. Be the first to share your experience with Late infantile CLN2 disease.

Start the conversation →

Latest news about Late infantile CLN2 disease

No recent news articles for Late infantile CLN2 disease.

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 Late infantile CLN2 disease

Are there clinical trials for Late infantile CLN2 disease?

Yes — 1 recruiting clinical trial is currently listed for Late infantile CLN2 disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Late infantile CLN2 disease?

5 specialists and care centers treating Late infantile CLN2 disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.