Leukodystrophy

Advocacy intelligence hub — real-time data for patient organizations

View full disease page →Search clinical trials →
Download CSV

At a Glance

Live from database

Regulatory Watchboard

8 events
Sep 2025An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

University of California, San Diego — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Sep 2024Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Massachusetts General Hospital — PHASE1, PHASE2

TrialENROLLING BY INVITATION
Mar 2024A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

Genetix Biotherapeutics Inc. — NA

TrialRECRUITING
Jul 2023A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

Minoryx Therapeutics, S.L. — PHASE3

TrialRECRUITING
Mar 2023An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

Calico Life Sciences LLC — PHASE1, PHASE2

TrialRECRUITING
Dec 2016The Myelin Disorders Biorepository Project

Children's Hospital of Philadelphia — NA

TrialRECRUITING
Sep 2014UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Joanne Kurtzberg, MD — PHASE1

TrialRECRUITING
Jan 2007Natural History, Diagnosis, and Outcomes for Leukodystrophies

University of Utah

TrialRECRUITING

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

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for Leukodystrophy.
Check the disease page for updates →

Clinical Trial Landscape

8 active trials
1Phase 3
1Phase 1
2N/A
1Unknown
3PHASE1, PHASE2
8Total recruiting
Search clinical trials for Leukodystrophy

Recent News & Research

No recent news articles indexed yet for Leukodystrophy.
Search PubMed for Leukodystrophy

Browse all Leukodystrophy news →

Specialist Network

Top 6 by expertise

View all Leukodystrophy specialists →

Quick Actions