Gene therapy for spinal muscular atrophy: Safety and early outcomes

  • Megan A. Waldrop
  • , Cassandra Karingada
  • , Mike A. Storey
  • , Brenna Powers
  • , Megan A. Iammarino
  • , Natalie F. Miller
  • , Lindsay N. Alfano
  • , Garey Noritz
  • , Ian Rossman
  • , Matthew Ginsberg
  • , Kathryn A. Mosher
  • , Eileen Broomall
  • , Jessica Goldstein
  • , Nancy Bass
  • , Linda P. Lowes
  • , Chang Yong Tsao
  • , Jerry R. Mendell
  • , Anne M. Connolly

Research output: Contribution to journal › Article › peer-review

107 Scopus citations

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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science