Ignite Creation Date:
2025-12-24 @ 11:02 PM
Ignite Modification Date:
2026-01-29 @ 9:14 PM
Study NCT ID:
NCT03047369
Status:
RECRUITING
Last Update Posted:
2025-10-23
First Post:
2017-02-01
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
The Myelin Disorders Biorepository Project
Sponsor:
Children's Hospital of Philadelphia
Organization:
Conditions & Keywords Data
Conditions:
Conditions
| Condition Brief | Condition Text | View |
|---|---|---|
| None | Leukodystrophy | View |
| None | White Matter Disease | View |
| None | Leukoencephalopathies | View |
| None | 4H Syndrome | View |
| None | Adrenoleukodystrophy | View |
| None | AMN | View |
| None | ALD | View |
| None | ALD Gene Mutation | View |
| None | ALD (Adrenoleukodystrophy) | View |
| None | X-linked Adrenoleukodystrophy | View |
| None | X-ALD | View |
| None | Adrenomyeloneuropathy | View |
| None | Aicardi Goutieres Syndrome | View |
| None | AGS | View |
| None | Alexander Disease | View |
| None | Alexanders Leukodystrophy | View |
| None | AxD | View |
| None | ADLD | View |
| None | Canavan Disease | View |
| None | CTX | View |
| None | Cerebrotendinous Xanthomatoses | View |
| None | Krabbe Disease | View |
| None | GALC Deficiency | View |
| None | Globoid Leukodystrophy | View |
| None | TUBB4A-Related Leukodystrophy | View |
| None | H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum | View |
| None | HBSL | View |
| None | HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity | View |
| None | LBSL | View |
| None | Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High … | View |
| None | Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation | View |
| None | ALSP | View |
| None | CSF1R Gene Mutation | View |
| None | HCC - Hypomyelination and Congenital Cataract | View |
| None | MLC1 | View |
| None | Megalencephalic Leukoencephalopathy With Subcortical Cysts | View |
| None | MLD | View |
| None | Metachromatic Leukodystrophy | View |
| None | PMD | View |
| None | Pelizaeus-Merzbacher Disease | View |
| None | PLP1 Null Syndrome | View |
| None | PLP1 Gene Duplication | Blood or Tissue | Mutations | View |
| None | Pelizaeus Merzbacher Like Disease | View |
| None | Peroxisomal Biogenesis Disorder | View |
| None | Zellweger Syndrome | View |
| None | Refsum Disease | View |
| None | Salla Disease | View |
| None | Sialic Storage Disease | View |
| None | Sjögren | View |
| None | Sjogren-Larsson Syndrome | View |
| None | Van Der Knapp Disease | View |
| None | Vanishing White Matter Disease | View |
| None | Charcot-Marie-Tooth | View |
| None | CMT | View |
| None | Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency | View |
| None | Allan-Herndon-Dudley Syndrome | View |
| None | Cadasil | View |
| None | Cockayne Syndrome | View |
| None | Multiple Sulfatase Deficiency | View |
| None | Gangliosidoses | View |
| None | GM2 Gangliosidosis | View |
| None | BPAN | View |
| None | Labrune Syndrome | View |
| None | LCC | View |
| None | Mucopolysaccharidoses | View |
| None | TBCK-Related Intellectual Disability Syndrome | View |