Spinal muscular atrophy (SMA) is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness. It is typically fatal and is commonly diagnosed in young children. Biogen's Spinraza, Novartis' Zolgensma, and Roche's Evrysdi are some of the approved therapies for SMA.
The company we are profiling today is Biohaven Ltd. (BHVN), a global clinical-stage biopharmaceutical company, approaching a significant milestone in the coming months related to its spinal muscular atrophy (SMA) trial.
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