PTC/GENZ:
Good thing besides they are still private - the drug is apparently safe and didn't alter 'normal' stop codons, which would result in off target AE. I would like to have answers to open questions such as:
Did full-length dystrophin appear in the muscles of the treated individuals?
Was the treatment more effective in individuals with the nonsense mutation TGA than TAA or TAG?
A lot can happen to change efficiency in other conditions: DNA sequence requirements, ribosome modifications, ribosomal protein variants... At this point I'd say that ataluren (PTC124) has a less reasonable chance to show meaningful physiological impact in nmDBMD without further modifications. However, its ability to promote ribosomal read-through of stop codons, might still show some level of activity that would translate to efficient treatment in other indications.
