Combination Therapy


Combination Therapy

SMA is a condition which, in addition to the motor neurons, affects other tissues and pathways. This means that it could be beneficial to address multiple aspects of the disease at the same time. 

Some pharmaceutical companies have started exploring this approach.

Trials of compounds that target the SMN2 gene and the muscle:


A clinical trial investigating the safety and efficacy of combining risdiplam, an SMN2 gene-enhancing molecule, with GYM329, an antimyostatin which enhances muscle growth and strength.

News article

TOPAZ (Scholar Rock)

Looks at the safety and efficacy of combining nusinersen (Biogen) with their muscle-enhancing product, SRK-015.

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