Gene therapy aims to halt fatal brain disease in children
NCT ID NCT03725670
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 10 participants, including infants as young as 1 month, will be monitored for safety and any changes in disease progression.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
lentiviral TYF-ARSA gene therapy
What this could lead to
If successful, this could slow or stop the progression of MLD, a severe genetic disorder, by correcting the underlying enzyme deficiency.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions or off-target effects.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Lung-Ji Chang
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••