Spinraza, the first treatment for the fatal genetic disease spinal muscular atrophy, offered hope that the same technology could transform other devastating neurological diseases. So far, progress has been slow

An­ti­sense tech­nol­o­gy changed one dev­as­tat­ing dis­ease. Why has­n't it trans­formed oth­ers?

The 2016 meet­ing of the Oligonu­cleotide Ther­a­peu­tics So­ci­ety in Mon­tre­al be­gan with a rare mix of promise and ela­tion.

That year, the 27-year-old biotech Io­n­is showed that a drug, lat­er brand­ed Spin­raza, could dra­mat­i­cal­ly al­ter the course of spinal mus­cu­lar at­ro­phy, a ge­net­ic con­di­tion that killed most di­ag­nosed chil­dren be­fore age 2. Io­n­is founder and CEO Stan­ley Crooke, in­tro­duced on stage by a video from past col­leagues and SMA pa­tients for a life­time achieve­ment award, was met with stand­ing ap­plause.

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