Seeking Alpha Seeking Alpha Portfolio App for iPad  Does it
matter how a drug works, if it works? PTC Therapeutics (PTCT)
seems bent on giving everyone an answer to that question,
because there sure seem to be a lot of questions about how
ataluren (PTC124), its Duchenne Muscular Dystrophy [DMD]
therapy, acts. This article [pdf] at Nature Biotechnology does
an excellent job explaining the details.  Premature "stop"
codons in the DNA of DMD patients, particularly in the
dystrophin gene, are widely thought to be one of the underlying
problems in the disease. (The same mechanism is believed to
operate in many other genetic-mutation-driven conditions as
well. Ataluren is supposed to promote "read-through" of these to
allow the needed protein to be produced anyway. That's not a
crazy idea at all - there's been a lot of thought about ways to
do that, and several aminoglycoside antibiotics have been shown
to work through that mechanism. Of that class, gentamicin has
been given several tries in the clinic, to ambiguou