The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.
|Slideshows|September 17, 2018
A Rare Genetic Disorder That Causes Episodes of Flaccid Paralysis
Author(s)Veronica Hackethal, MD
About 30% of patients with periodic paralysis have unknown genetic mutation, making identification and treatment a challenge. Here: key points on genetics, testing, and subtypes of PPP.
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