SMA Moment: Will Gene Therapy Shift Treatment, Costs of Muscle Disease?
Summary
This is a big moment for people diagnosed with spinal muscular atrophy, or SMA, a rare and potentially lethal genetic disorder that destroys muscles. A recent survey of 30 physicians in the US and Europe by the investment bank Jefferies suggested that a majority of newly diagnosed SMA patients, as well as those currently on Spinraza, will get Zolgensma. Alex Fay, a pediatric neurologist at UCSF Benioff Children’s Hospital in San Francisco, CA, says he would be hesitant to switch patients if Spinraza is well tolerated and working. Citing the study and other data supporting Spinraza, Biogen CEO Michel Vounatsos was adamant on an April 24 conference call that the drug “will remain the standard of care for SMA for years to come.” The presentations this weekend will shed more light on the potential benefits and risks of the new world of SMA treatments, but there will plenty of questions left unanswered.
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