This very well-written article will help a lot of investors learn how to separate the wheat from the chaff as far as gene therapy companies go.
One (misleading) point he makes really only applies to our current shortened DMD molecule...Our LGMD drugs use the original full-length gene. He seems to think all our gene therapy molecules are shortened versions. Not so.
"It's also important for investors to acknowledge one unaddressed drawback: MyoAAV delivery shuttles will still have a relatively low capacity. That will force Sarepta Therapeutics to continue using miniaturized versions of replacement genes instead of full-length, human copies. Higher delivery efficiency should reduce the impact of this limitation, but the drawback looms."