Adult-onset autosomal dominant leukodystrophy

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ORPHA:99027OMIM:169500E75.2
Who is this for?
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1Active trials10Specialists8Treatment centers

Where are you in your journey?

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

Also known as:

ADLDAdult-onset autosomal dominant demyelinating leukodystrophy

Clinical phenotype terms— hover any for plain English:

Autonomic bladder dysfunctionHP:0005341Atrophy of the spinal cordHP:0006827Abnormal auditory evoked potentialsHP:0006958Atrophy/Degeneration affecting the brainstemHP:0007366Abnormality of somatosensory evoked potentialsHP:0007377Abnormal cerebellar peduncle morphologyHP:0011931
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Mar 2025Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

n-Lorem Foundation — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Adult-onset autosomal dominant leukodystrophy.

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

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →

Specialists

10 foundView all specialists →
JZ
Jin-Long Zou
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
SH
Shuang He
OAKLAND GARDENS, NY
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
JZ
Jie-Wen Zhang
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
SL
Shu-Jian Li
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
IM
Izumi Mihashi
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
KI
Kazuhiro Ishii
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
NH
Norikazu Hara
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
TI
Takeshi Ikeuchi
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
SS
Shinji Saiki
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication
SC
Shuai Chen
BROOKLYN, NY
Specialist
1 Adult-onset autosomal dominant leukodystrophy publication

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 Adult-onset autosomal dominant leukodystrophy.

Search all travel grants →NORD Financial Assistance ↗

Community

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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 Adult-onset autosomal dominant leukodystrophy

Are there clinical trials for Adult-onset autosomal dominant leukodystrophy?

Yes — 1 recruiting clinical trial is currently listed for Adult-onset autosomal dominant leukodystrophy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Adult-onset autosomal dominant leukodystrophy?

10 specialists and care centers treating Adult-onset autosomal dominant leukodystrophy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.