Search Results - "Lehman, Kelly J."

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations by Mendell, Jerry R., Connolly, Anne M., Lehman, Kelly J., Griffin, Danielle A., Khan, Sohrab Z., Dharia, Sachi D., Quintana-Gallardo, Lucía, Rodino-Klapac, Louise R.

“…Given the increasing number of gene transfer therapy studies either completed or underway, there is growing attention to the importance of preexisting adaptive…”
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Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy by Mendell, Jerry R, Shieh, Perry B, McDonald, Craig M, Sahenk, Zarife, Lehman, Kelly J, Lowes, Linda P, Reash, Natalie F, Iammarino, Megan A, Alfano, Lindsay N, Sabo, Brenna, Woods, Jeremy D, Skura, Christy L, Mao, Howard C, Staudt, Loretta A, Griffin, Danielle A, Lewis, Sarah, Wang, Shufang, Potter, Rachael A, Singh, Teji, Rodino-Klapac, Louise R

“…Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy designed for targeted expression of SRP-9001 dystrophin protein, a…”
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Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy by Mendell, Jerry R, Al-Zaidy, Samiah A, Lehman, Kelly J, McColly, Markus, Lowes, Linda P, Alfano, Lindsay N, Reash, Natalie F, Iammarino, Megan A, Church, Kathleen R, Kleyn, Aaron, Meriggioli, Matthew N, Shell, Richard

“…This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene…”
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Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy by Lowes, Linda P., Alfano, Lindsay N., Arnold, W. David, Shell, Richard, Prior, Thomas W., McColly, Markus, Lehman, Kelly J., Church, Kathleen, Sproule, Douglas M., Nagendran, Sukumar, Menier, Melissa, Feltner, Douglas E., Wells, Courtney, Kissel, John T., Al-Zaidy, Samiah, Mendell, Jerry

“…This study characterizes motor function responses after early dosing of AVXS-101 (onasemnogene abeparvovec) in gene replacement therapy in infants with severe…”
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Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial by Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle A., Hogan, Mark, Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino‐Klapac, Louise R.

“…Introduction/Aims Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with…”
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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) by 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.

“…Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR…”
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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results by Mendell, Jerry R., Pozsgai, Eric R., Lewis, Sarah, Griffin, Danielle A., Lowes, Linda P., Alfano, Lindsay N., Lehman, Kelly J., Church, Kathleen, Reash, Natalie F., Iammarino, Megan A., Sabo, Brenna, Potter, Rachael, Neuhaus, Sarah, Li, Xiaoxi, Stevenson, Herb, Rodino-Klapac, Louise R.

“…Limb-girdle muscular dystrophy 2E/R4 is caused by mutations in the β-sarcoglycan ( SGCB ) gene, leading to SGCB deficiency and consequent muscle loss. We…”
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Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing by Mah, May Ling, Cripe, Linda, Slawinski, Michelle K., Al-Zaidy, Samiah A., Camino, Eric, Lehman, Kelly J., Jackson, Jamie L., Iammarino, Megan, Miller, Natalie, Mendell, Jerry R., Hor, Kan N.

“…Varied detection methods have resulted in conflicting reports on the prevalence of cardiac disease in Duchenne and Becker muscular dystrophy carriers (MDC). We…”
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Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age Group–Matched Controls by Jackson, Jamie L., Korth, Christina X., Leslie, Carine E., Cotto, Jennifer, Mah, May Ling, Hor, Kan, Cripe, Linda, Al-Zaidy, Samiah, Camino, Eric M., Church, Kathleen, Lehman, Kelly J., Shay, Victoria, Mendell, Jerry R.

“…The health-related quality of life and emotional distress among mothers of sons with Duchenne or Becker muscular dystrophies (n = 82) were compared to sex- and…”
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P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study by O’Rourke, Erin, Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan, Alfano, Lindsay, Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle, Hogan, Mark J., Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino-Klapac, Louise

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Safety, β-Sarcoglycan Expression, and Functional Outcomes From Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4 (S23.005) by Rodino-Klapac, Louise R., Pozsgai, Eric R., Lewis, Sarah, Griffin, Danielle A., Meadows, Aaron S., Lehman, Kelly J., Church, Kathleen, Reash, Natalie F., Iammarino, Megan A., Powers, Brenna, Alfano, Lindsay N., Lowes, Linda P., Koenig, Erica, Neuhaus, Sarah, Li, Xiaoxi, Mendell, Jerry R.

“…Abstract only…”
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Phase 1/2a Trial of SRP-9001 in Patients with Duchenne Muscular Dystrophy: 3-Year Safety and Functional Outcomes (S23.004) by Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Nease, Carrie, Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Vaiea, Jordan, Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle A., Pozsgai, Eric R., Hogan, Mark, Hu, Larry, Giblin, Kathryn, Rodino-Klapac, Louise R.

“…Abstract only…”
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A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of SRP-9001 for Treating Patients with Duchenne Muscular Dystrophy (S23.002) by Mendell, Jerry R., Shieh, Perry B., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Powers, Brenna, Woods, Jeremy D., Skura, Christy L., Mao, Howard C., Staudt, Loretta A., Potter, Rachael A., Griffin, Danielle A., Lewis, Sarah, Hu, Larry, Upadhyay, Sameer, Singh, Teji, Rodino-Klapac, Louise R.

“…Abstract only…”
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AVXS-101 Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Phase 1 Clinical Trial (S25.006) by Mendell, Jerry R., Lehman, Kelly J., McColly, Markus, Lowes, Linda P., Alfano, Lindsay N., Miller, Natalie F., Iammarino, Megan A., Church, Kathleen, Ogrinc, Francis G., L’Italien, James, Wells, Courtney, Sproule, Douglas M., Feltner, Douglas E.

“…Abstract only…”
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Gene-Replacement Therapy in Spinal Muscular Atrophy Type 1: Long-Term Follow-Up From the Onasemnogene Abeparvovec-xioi Phase 1/2a Clinical Trial (1808) by Mendell, Jerry R., Shell, Richard, Lehman, Kelly J., McColly, Markus, Lowes, Linda P., Alfano, Lindsay N., Miller, Natalie F., Iammarino, Megan A., Church, Kathleen, Ogrinc, Francis G., Ouyang, Haojun, Kernbauer, Elaine, Joshi, Shivani, Sproule, Douglas M., Meriggioli, Matthew, Feltner, Douglas E., Al-Zaidy, Samiah

“…Abstract only…”
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LATE BREAKING NEWS E-POSTER PRESENTATION by Willcocks, Dr. Rebecca J, Forbes, Dr. Sean C, Lott, Dr. Donovan J, Senesac, Dr. Claudia R, Barnard, Dr. Alison M, Lehman, Kelly J, Nease, Carrie E, Church, Kathleen, Sahenk, Dr. Zarife, Sweeney, Dr. H. Lee, Rodino-Klapac, Dr. Louise R, Walter, Dr. Glenn A, Mendell, Dr. Jerry R, Vandenborne, Dr. Krista

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LATE BREAKING NEWS E-POSTER PRESENTATION: LBP 13 rAAVrh.74.MHCK7.micro-dystrophin gene therapy in children with Duchenne muscular dystrophy: A quantitative MR biomarker study by Willcocks, Dr. Rebecca J, Forbes, Dr. Sean C, Lott, Dr. Donovan J, Senesac, Dr. Claudia R, Barnard, Dr. Alison M, Lehman, Kelly J, Nease, Carrie E, Church, Kathleen, Sahenk, Dr. Zarife, Sweeney, Dr. H. Lee, Rodino-Klapac, Dr. Louise R, Walter, Dr. Glenn A, Mendell, Dr. Jerry R, Vandenborne, Dr. Krista

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