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Comment
. 2017 Oct 4;25(10):2235-2237.
doi: 10.1016/j.ymthe.2017.09.002. Epub 2017 Sep 8.

Unfounded Claims of Improved Functional Outcomes Attributed to Follistatin Gene Therapy in Inclusion Body Myositis

Steven A Greenberg  1
Affiliations
Comment

Unfounded Claims of Improved Functional Outcomes Attributed to Follistatin Gene Therapy in Inclusion Body Myositis

Steven A Greenberg. Mol Ther. .
No abstract available

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Figures

Figure 1
Figure 1
Study Design Does Not Allow for Conclusions of Efficacy for Follistatin Gene Therapy (A) Study design and its relationship to clinical trial NCT01519349. (B) Hypothetical design in which 3 of the 4 interventions are controlled through blinding and use of a comparator group. 6MWT, 6-min walk test
See this image and copyright information in PMC

Comment in

  • Reply to Letter to the Editor.
    Mendell JR. Mendell JR. Mol Ther. 2017 Oct 4;25(10):2238-2240. doi: 10.1016/j.ymthe.2017.09.003. Epub 2017 Sep 8. Mol Ther. 2017. PMID: 28939086 Free PMC article. No abstract available.

Comment on

  • Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
    Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, Lewis S, McColly M, Hogan MJ, Kaspar BK. Mendell JR, et al. Mol Ther. 2017 Apr 5;25(4):870-879. doi: 10.1016/j.ymthe.2017.02.015. Epub 2017 Mar 6. Mol Ther. 2017. PMID: 28279643 Free PMC article.

References

    1. Mendell J.R., Sahenk Z., Al-Zaidy S., Rodino-Klapac L.R., Lowes L.P., Alfano L.N., Berry K., Miller N., Yalvac M., Dvorchik I. Follistatin gene therapy for sporadic inclusion body myositis improves functional outcomes. Mol. Ther. 2017;25:870–879. - PMC - PubMed
    1. Mendell, J.R. (2017). Follistatin Gene Transfer to Patients with Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis. https://clinicaltrials.gov/ct2/show/NCT01519349.
    1. Mendell J.R., Sahenk Z., Malik V., Gomez A.M., Flanigan K.M., Lowes L.P., Alfano L.N., Berry K., Meadows E., Lewis S. A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy. Mol. Ther. 2015;23:192–201. - PMC - PubMed
    1. Johnson L.G., Edwards D.J., Walters S.E., Thickbroom G.W., Mastaglia F.L. The effectiveness of an individualized, home-based functional exercise program for patients with sporadic inclusion body myositis. J. Clin. Neuromuscul. Dis. 2007;8:187–194.
    1. Ahmed M., Machado P.M., Miller A., Spicer C., Herbelin L., He J., Noel J., Wang Y., McVey A.L., Pasnoor M. Targeting protein homeostasis in sporadic inclusion body myositis. Sci. Transl. Med. 2016;8:331ra41. - PMC - PubMed