Its flagship project was intepirdine, an experimental 5-HT6 receptor antagonist it acquired ... deals involving a pair of gene therapies for GM1 and GM2 gangliosidosis – AXO-AAV-GM1 and AXO ...
Ophthalmology: ptosis (mitochondrial disorders, Menkes syndrome), retinal degeneration (grey matter disease), optic atrophy (white matter disease), cherry-red spots (GM1/2 gangliosidosis ...
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