Novartis Stopping Work on Branaplam as Oral SMA Therapy

Novartis Stopping Work on Branaplam as Oral SMA Therapy

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Novartis Stopping Work on Branaplam as Oral SMA Therapy

Novartis is stopping the clinical development of branaplam (LMI070), its experimental oral treatment for spinal muscular atrophy (SMA) being evaluated in a Phase 1/2 clinical trial. According to the company’s announcement, this “difficult” decision was based on the “rapid advancements in the SMA treatment landscape in recent years,” and the fact that branaplam would “not offer a highly differentiated treatment solution for the SMA community.” Within four years, three disease-modifying therapies became available for SMA in…

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