A recent Phase I/II clinical study led by UMass Chan Medical School has shown encouraging results for a gene therapy targeting GM2 gangliosidosis, a group of rare and fatal neurodegenerative…
Early clinical data from a Phase I/II study at UMass Chan Medical School point to meaningful, measurable benefits from a central nervous system gene therapy for GM2 gangliosidosis, which encompasses…
Within the United States, Orphan Drug designation is granted by the FDA to facilitate the development and review of drugs or biologics intended to treat rare conditions. In this…
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