After 30 years with nothing, a medicine for Alexander disease

For families facing Alexander disease — an ultra-rare disorder striking fewer than one in a million people, attacking the brain's insulation and stealing movement, speech, even breathing — there was never anything but symptom management. That changed with the approval of Zanvastro, the first drug that targets the disease's underlying cause. In trials, patients taking it for just over a year walked significantly faster than untreated patients, and motor function stabilized or improved.