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Pharmaceutical company PTC Therapeutics recently published the latest data from a key FIREFISH study that evaluated the effectiveness and safety of Evrysdi™ (risdiplam) in the treatment of type 1 spinal muscular atrophy (SMA) infants.
21 children recruited in part 1 of the FIREFISH study.
infection
The pharmacodynamic data showed that the SMN protein of the patients in the Evrysdi treatment group increased by 1.
The SMN protein of the patients in the Evrysdi treatment group increased by 1.
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