Chemical structure of molecule HTT-D3

HTT splicing modulator

PD in Huntington’s disease model

from HTS and optimization

Nature Communications

PTC Therapeutics, So. Plainfield, NJ

Mutated Huntingtin (HTT) protein is the cause of Huntington’s disease (HD). HTT-D3 is an oral, CNS-penetrant molecule with human HTT lowering activity in vivo used to illustrate the science…

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