Research Topics


Marla Weetall


Affiliation: PTC Therapeutics
Country: USA


  1. Zhao X, Feng Z, Ling K, Mollin A, Sheedy J, Yeh S, et al. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy. Hum Mol Genet. 2016;25:1885-1899 pubmed
    ..Overall, we provide important insights for the translation of pre-clinical data to the clinic and further therapeutic development of this series of molecules for SMA treatment. ..
  2. Feng Z, Ling K, Zhao X, Zhou C, Karp G, Welch E, et al. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset. Hum Mol Genet. 2016;25:964-75 pubmed publisher