Rare diseases
Investigational molecules
Locations
17 State Street, 10004-1501, United States
Research activity is centered on Canavan disease, a rare inherited leukodystrophy affecting the central nervous system and early brain development. The sponsor’s clinical focus reflects interest in conditions driven by defects in metabolic pathways linked to neurological injury.
The therapeutic emphasis is on a gene therapy approach aimed at addressing the underlying biology of this disorder in children.
Myrtelle Inc. runs clinical trials in conditions such as Canavan disease, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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