SMA therapy: paving the way for drug development strategies

Alessia Deglincerti (pictured above, left), Frank Borowsky and Rachel Laing from consulting firm Bionest Partners, explore in an Expert View how the development of spinal muscular atrophy (SMA) therapies could provide a blueprint for genetic diseases.

Disease-modifying therapeutics have vigorously entered the market for the treatment of SMA, a rare sight for neurological and neuromuscular diseases.

The development of these SMA drugs not only brings new opportunities for additional, supportive drugs as a new SMA disease progression emerges, but also provides companies with a blueprint for franchise, commercial, and payer strategies that can be translated to other diseases where next-generation therapies will also become the new standard of care.