The Ionis drug is pretty much the same as the Ultragenyx drug (an ASO aimed at releasing paternal UBE3A silencing), so perhaps not surprising it has yielded similar results.
It therefore suffers from the same limitations discussed above, particularly the need for repeated administration by intrathecal injection. NNZ-2591 has a significant advantage being delivered orally if it can match or exceed efficacy.
The Ionis drug is pretty much the same as the Ultragenyx drug...
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