Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA
Lingling Kong(Johns Hopkins University), David Valdivia(Johns Hopkins University), Christian M. Simon(Columbia University), Cera W. Hassinan(Johns Hopkins University), Nicolas Delestrée(Columbia University), Daniel M. Ramos(Johns Hopkins University), Jae Hong Park(Johns Hopkins University), Celeste M. Pilato(Johns Hopkins University), Xixi Xu(Johns Hopkins University), Melissa Crowder(Johns Hopkins University), Chloe C. Grzyb(Johns Hopkins University), Zachary A. King(Johns Hopkins University), Marco Petrillo(Biogen (United States)), Kathryn J. Swoboda(Harvard University), Crystal Davis(Jackson Laboratory), Cathleen Lutz(Jackson Laboratory), Alexander Stephan(Roche (Switzerland)), Xin Zhao(PTC Therapeutics (United States)), Marla Weetall(PTC Therapeutics (United States)), Nikolai A. Naryshkin(PTC Therapeutics (United States)), Thomas O. Crawford(Johns Hopkins University), George Z. Mentis(Columbia University), Charlotte J. Sumner(Johns Hopkins University)
Cited by 126Open Access
Abstract
splice modifiers beginning immediately after birth in mice increased radial growth of the already myelinated axons, but in utero treatment was required to restore axonal growth and associated maturation, prevent subsequent neonatal axon degeneration, and enhance motor axon function. Together, these data reveal a cellular basis for the fulminant neonatal worsening of patients with infantile onset SMA and identify a temporal window for more effective treatment. These findings suggest that minimizing treatment delay is critical to achieve optimal therapeutic efficacy.
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