Central nervous system malformation

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ORPHA:98044
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38Specialists8Treatment centers

Where are you in your journey?

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

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
May 2026The Association Between Tau & GFAP and Postoperative Neurological Impairment in Patients Undergoing Cardiac Surgery

Boston Children's Hospital

TrialNOT YET RECRUITING
Apr 2026Comparing Two PFO Closure Devices in Adults With Previous Stroke or TIA

Giuseppe Tarantini — NA

TrialNOT YET RECRUITING
Oct 2025Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study

Barboni

TrialNOT YET RECRUITING
Sep 2025Structural Validity and Inter-rater Reliabitiliy of the Ataxia Trunk, Lower And Upper Extremity Scale (ATLAS)

Haute Ecole de Santé Vaud

TrialNOT YET RECRUITING
Jun 2025Stemchymal® for Polyglutamine Spinocerebellar Ataxia

Steminent US, Inc. — PHASE2

TrialNOT YET RECRUITING
May 2025SeizEAR Safety Study

Icahn School of Medicine at Mount Sinai — NA

TrialRECRUITING
Mar 2025Efficacy of Cerebellar Transcranial Magnetic Stimulation to Treat Hereditary Spinocerebellar Ataxias

Xijing Hospital — NA

TrialNOT YET RECRUITING
Feb 2025Percutaneous Repair for Drug - Resistant Epilepsy by Intervention of Closing the Patent Foramen Ovale（PREDICT-PFO Trial）

Sichuan University — NA

TrialRECRUITING
Oct 2024Enhancement of Quality of Work And Life

Radboud University Medical Center — NA

TrialRECRUITING
Jun 2024Medical Treatment With or Without Transcatheter Patent Foramen Ovale Closure

Josep Rodes-Cabau — NA

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Central nervous system malformation.

View clinical trials →

Clinical Trials

View all trials with filters →

No actively recruiting trials found for Central nervous system malformation at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Central nervous system malformation community →

Specialists

Showing 25 of 38View all specialists →
ZY
Zhiheng Yan
Specialist
1 Central nervous system malformation publication
XZ
Xia Zhang
Specialist
1 Central nervous system malformation publication
XX
Xiaoyan Xu
Specialist
1 Central nervous system malformation publication
BP
Bo Pang
HOUSTON, TX
Specialist
1 Central nervous system malformation publication
SB
Sumeet Bhardwaj
LINCOLN, CA
Specialist
1 Central nervous system malformation publication
YW
Yixuan Wang
BURNSVILLE, MN
Specialist
1 Central nervous system malformation publication
AK
Aayush Kapoor
Specialist
1 Central nervous system malformation publication
QL
Quanlin Li
Specialist
1 Central nervous system malformation publication
YL
Yan Lu
Specialist
1 Central nervous system malformation publication
LY
Lei Yang
Specialist
1 Central nervous system malformation publication
BM
Bin Ma
CALDWELL, ID
Specialist
1 Central nervous system malformation publication
TL
Tiangang Li
Specialist
1 Central nervous system malformation publication
YX
Yuan Xie
CAPE GIRARDEAU, MO
Specialist
1 Central nervous system malformation publication
JP
Jing-Jing Pan
Specialist
1 Central nervous system malformation publication
QL
Qin Li
Specialist
1 Central nervous system malformation publication
TM
Tippi C MacKenzie, MD
San Francisco, California
Specialist

Rare Disease Specialist

PI on 2 active trials
PM
Prashant Chittiboina, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 7 active trials
PM
Paul S. Aisen, M.D.
SAN DIEGO, CA
Specialist
PI on 2 active trials
AM
Anita MacDonald
Birmingham
Specialist

Rare Disease Specialist

PI on 10 active trials
MM
Marc Kamin, MD
OYSTER BAY, NY
Specialist
PI on 3 active trials
SP
Steven M Hersch, MD, PhD
BOSTON, MA
Specialist
PI on 2 active trials
DM
Debra J Ehrlich, M.D.
GLENDALE, AZ
Specialist
PI on 3 active trials
HP
Huma Arshad Cheema, Prof.
Lahore, NY
Specialist

Rare Disease Specialist

PI on 1 active trial
DM
Don Nwose, MD
Specialist
PI on 2 active trials
SP
Sergey Stavisky, Ph.D.
Specialist
PI on 1 active trial

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 Central nervous system malformation.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Central nervous system malformationForum →

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Latest news about Central nervous system malformation

Disease timeline:

New recruiting trial: GMP Powdered Substitutes in PKU and TYR

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Comparative Outcomes Related to Delivery-room Cord Milking In Low-resourced Kountries Developmental Follow Up

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Functional Imaging of Baby Brains

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Medical Treatment With or Without Transcatheter Patent Foramen Ovale Closure

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Enhancement of Quality of Work And Life

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: SeizEAR Safety Study

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Phenotype/Genotype Correlations in Movement Disorders

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Percutaneous Repair for Drug - Resistant Epilepsy by Intervention of Closing the Patent Foramen Ovale（PREDICT-PFO Trial）

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Physiological Assessments During Non Operative Treatment on the Chest Wall Deformities

A new clinical trial is recruiting patients for Central nervous system malformation

New recruiting trial: Ocular Characteristics in Patients With Craniosynostosis

A new clinical trial is recruiting patients for Central nervous system malformation

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 Central nervous system malformation

Which specialists treat Central nervous system malformation?

25 specialists and care centers treating Central nervous system malformation are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.