Yes, they would qualify as orphan indications and potentially benefit from NNZ-2591 but prevalence seems to be quite low.
For example, for trisomy 18, only 5- 10% of babies born with the condition survive beyond 12 months. The survival rate seems to be higher for monosomy 18p but prevalence is still lower than for Pitt Hopkins syndrome.
Yes, they would qualify as orphan indications and potentially...
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