5 Experimental SMA Therapies You Might Find Interesting


4. RG7800

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PTC’s more developed SMA drug candidate, RG7800, is the subject of a Phase 2 trial in adult and pediatric SMA patients, but dosing was suspended and the trial was placed on clinical hold to investigate a non-clinical safety finding observed in a longer-term animal study.

Learn more about how RG7800 can be used to treat SMA.


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