Clinical trials: Giant Axonal Neuropathy
Giant Axonal Neuropathy, Gene Transfer
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly.
- Trial name
- NCT02362438
- Testing
- scAAv9/JeT-GAN
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Giant Axonal Neuropathy (GAN)
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous…
- Trial name
- NCT07543991
- Testing
- scAAV9/JeT-GAN
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Giant Axonal Neuropathy
Giant Axonal Neuropathy Natural History Study
Giant Axonal Neuropathy (GAN) is a devastating and rare childhood disease. Children with GAN develop increasing muscle weakness, impaired sensation, and at times mental retardation.
- Trial name
- NCT01503125
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Columbia University