Start Making Sense - Exon Skipping & Nonsense Suppression
Final thoughts
It is a bitter reflection that the initial excitement on the sequencing of the dystrophin gene did not manifest itself in a treatment in time to allow many of those born with DMD in that year to reach their coming-of-age 21 years later. However, the outlook for these more creative approaches makes the picture rather more hopeful for the current generation. And whatever the outcome for muscular dystrophy, it will certainly have profound implications regarding treatments for the entire spectrum of genetic disorders. Perhaps the one-pill panacea is not so far away as we might have thought.
Janet Beal
Biotech Editor
Pharmaprojects - Pharmaceutical Research & Development Pipeline Intelligence
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