SMA THERAPIES II AND BIOMARKERS

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Branaplam is an orally bioavailable molecule that interacts with SMN2 RNA to increase functional survival motor neuron protein and is being developed for treatment of SMA. This open-label, multi-part, first-in-human study is evaluating the safety and efficacy of branaplam in patients with SMA type I. The study enrolled 14 patients, 1 failed screening, and 13 received once-weekly branaplam with planned dose escalation based on safety results. All patients continued from an initial 13-week treatment period into a long-term extension.

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