A proof-of-concept study of growth hormone in children with Phelan-McDermid syndrome.
Swathi SethuramTess LevyJennifer H Foss-FeigDanielle HalpernSven SandinPaige M SiperH WalkerJoseph D BuxbaumRobert RapaportAlexander KolevzonPublished in: Molecular autism (2022)
Overall, findings are promising and indicate the need for larger studies with rhGH in PMS. Trial registration NCT04003207. Registered July 1, 2019, https://clinicaltrials.gov/ct2/show/NCT04003207 .