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4050 Branaplam
4c36d6b14cbcf331a500fe52b9535e532ab3ba1b252e2959b73a464b0d15d58a

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Properties

Chemistry

Branaplam (development codes LMI070 and NVS-SM1) is an experimental drug being developed by Novartis to treat spinal muscular atrophy (SMA). It is a pyridazine derivative that works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.

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