Alnylam, a start-up drug company, expects soon to have on the market a drug treating familial amyloid polyneuropathy, which is rare and presently incurable. The disease destroys motor skills and most people stricken by it die within 10 to 15 years. That is wonderful news for those with the disease and for their families. But it’s actually much better news that that.
The drug is unique in using a recently discovered technique to disable the gene causing the disease. If this works as it appears it will, then many treatments for many more common ailments may soon be on the way. This is fantastic news for all of us.