Tominersen

Harmony Biosciences receives FDA Refusal to File letter for pitolisant's supplemental New Drug Application in adult idiopathic hypersomnia patients with excessive daytime sleepiness.

uniQure's gene therapy trial has shown promising results, with treated patients exhibiting a significant reduction in the rate of Huntington's disease progression.

Vico Therapeutics' VO659, an antisense oligonucleotide, targets CAG repeats in Huntington's disease (HD) and spinocerebellar ataxias (SCA1, SCA3).

Novartis and PTC Therapeutics have entered into a collaboration for the development and commercialization of PTC518, a potential treatment for Huntington's disease.

Several companies are advancing potential Huntington's disease treatments, including Wave Life Sciences with WVE-003 and uniQure with AMT-130.