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doi: 10.1007/978-3-030-89340-8_3 19 AgarwalASharmaRKSharmaRAssidiMAbuzenadahAMAlshahraniSet al

These two pathways converge at the somatotroph and produce a GH pulse that neither compound achieves alone

Although it has been suggested that further clinical trials with a patient placebo-controlled arm are required to truly confirm the efficacy of this gene therapy, this can be challenging in rare diseases like LHON