[1][2] In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases.

[8] In 2018, PTC acquired Agilis Biotherapeutics and a gene therapy candidate, GT-AADC, with its compelling clinical data in treating aromatic L-amino acid decarboxylase (AADC) deficiency.

[12] In 2020, PTC announced the FDA approval of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older.

[14] PTC Therapeutics’ therapy, known as PTC518, is an oral medication designed to reduce the production of the mutant protein linked to the genetic mutation responsible for the disease.

[16] In June, interim data from a Phase 2 trial indicated that PTC518 successfully lowered levels of the mutant protein in both blood and cerebrospinal fluid.