The drug is the first and only treatment that can change the course of this rare neurological disease. For decades, patients diagnosed with Alexander disease had no options beyond managing their symptoms. The ultra-rare, progressive, and often fatal neurological disorder affects fewer than one in a million people worldwide. It affects motor and cognitive abilities, [...] FDA Approves First Drug To Treat Alexander Disease After 30 Years of Research
The drug is the first and only treatment that can change the course of this rare neurological disease. For decades, patients diagnosed with Alexander disease had no options beyond managing their symptoms. The ultra-rare, progressive, and often fatal neurological disorder affects fewer than one in a million people worldwide. It affects motor and cognitive abilities, [...] 

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