Sarepta's CMT therapy - not a "different sub-type" ....
...rather a substitute created protein that does the CMT protein's job ... as well as a whole lot more - it can tackle a whole lot of other, more common, conditions, as I recall.
Just like our GALGT2 program. They (CMT and GALGT2) "officially" target one named disease, but actually target a slew. And, parenthetically, this could be what we come up with with the contributions that could be coming from our Florida colleagues. For example, a combo therapy for Parkinson's, Alzheimer's and a bunch of other related things - all handled by the same or a very similar combo therapy.
So, a lot of greenhorn biotechs are popping up out of nowhere (like this CMT one called Passages Biotech or some such) with a GT therapy for a sub-type of Whatever - where one exon is at fault and they fix it (like our various LGMD things).
And then there are the long-time players in the field of genetic medicine (Sarepta comes to mind) who can see more clever - elegant - things that can be done and that will kill MANY different "birds" with one silver (or Gold) bullet and/or stone.