Extended Data Fig. 4: PQ-110-001 trial: Schema for introduction of dose levels. | Nature Medicine

Extended Data Fig. 4: PQ-110-001 trial: Schema for introduction of dose levels.

From: Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

Extended Data Fig. 4

The safety monitoring committee and/or Sponsor Medical Monitor could decide to de-escalate the dose, hold the dose (delay or skip), or discontinue study drug for an individual subject, in consultation with the Investigator. Subjects who discontinued study drug continued to be followed for safety and efficacy. A = adult subjects; DMC = Data Monitoring Committee; P = pediatric subjects.

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