Novartis AG has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of familial dysautonomia, Huntington’s disease and spinal muscular atrophy.
Biogen Inc. has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of Huntington’s disease and spinal muscular atrophy (SMA).