Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series von Chand, Deepa H., Zaidman, Craig, Arya, Kapil, Millner, Rachel, Farrar, Michelle A., Mackie, Fiona E., Goedeker, Natalie L., Dharnidharka, Vikas R., Dandamudi, Raja, Reyna, Sandra P.

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Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy von Goedeker, Natalie L., Dharia, Sachi D., Griffin, Danielle A., Coy, Jesantha, Truesdale, Todd, Parikh, Rajan, Whitehouse, Kasen, Santra, Sourav, Asher, Damon R., Zaidman, Craig M.

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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR)... von Zaidman, Craig M., Proud, Crystal M., McDonald, Craig M., Lehman, Kelly J., Goedeker, Natalie L., Mason, Stefanie, Murphy, Alexander P., Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle A., Potter, Rachael A., Rodino‐Klapac, Louise R., Mendell, Jerry R.

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Laboratory monitoring of nusinersen safety von Goedeker, Natalie L., Gibbons, Jennifer L., Varadhachary, Arun S., Connolly, Anne M., Zaidman, Craig M.

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Outcomes of early‐treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study von Goedeker, Natalie L., Rogers, Amanda, Fisher, Mark, Arya, Kapil, Brandsema, John F., Farah, Hiba, Farrar, Michelle A., Felker, Marcia V., Gibbons, Melissa, Hamid, Omer Abdul, Harmelink, Matthew, Herbert, Karen, Kichula, Elizabeth, King, Kiana, Lakhotia, Arpita, Lee, Bo Hoon, Kuntz, Nancy L., Parsons, Julie, Rehborg, Rebecca, Veerapaniyan, Aravindhan, Zaidman, Craig M.

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Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States von Zaidman, Craig M, Crockett, Cameron D, Wedge, Ethan, Tabatabai, Grace, Goedeker, Natalie

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Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy von Zaidman, Craig M., Goedeker, Natalie L., Aqul, Amal A., Butterfield, Russell J., Connolly, Anne M., Crystal, Ronald G., Godwin, Kara E., Hor, Kan N., Mathews, Katherine D., Proud, Crystal M., Kula Smyth, Elizabeth, Veerapandiyan, Aravindhan, Watkins, Paul B., Mendell, Jerry R.

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Evaluation of Total Binding Antibodies Against rAAVrh74 in Patients with Duchenne Muscular Dystrophy (P14-13.005) von Goedeker, Natalie L., Griffin, Danielle, Dharia, Sachi, Santra, Sourav, Coy, Jesantha, Yocum, Nicole, Zaidman, Craig M.

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Clinical Trial Experiences Managing Adverse Events in Gene Therapy for DMD: Natalie L. Goedeker, CPNP von Goedeker, Natalie L

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