Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu-Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)768-782
Number of pages15
JournalJournal of Cachexia, Sarcopenia and Muscle
Issue number3
Early online date7 Feb 2020
Publication statusPublished - Jun 2020


  • Adeno-associated virus
  • Antisense oligonucleotide
  • Combinatorial therapy
  • Myostatin inhibition
  • Myostatin propeptide
  • Spinal muscular atrophy
  • Survival of motor neuron 1 gene

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