Search Results - "Reash, Natalie F."

Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study by Iammarino, Megan A, Alfano, Lindsay N, Reash, Natalie F, Sabo, Brenna, Conroy, Sara, Noritz, Garey, Wendland, Madalynn, Lowes, Linda P

“…This single-arm prospective cohort study aimed to evaluate the feasibility and utility of in-home body weight support harness system (BWSS) use in children…”
Get full text

Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy by P Lowes, Linda, Alfano, Lindsay N, Iammarino, Megan A, Reash, Natalie F, Giblin, Kathryn, Hu, Larry, Yu, Lixi, Wang, Shufang, Salazar, Rachel, Mendell, Jerry R

“…Conducting functional assessments remotely can help alleviate the burden of in-person assessment on patients with Duchenne muscular dystrophy and their…”
Get full text

Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic by James, Meredith K., Rose, Kristy, Alfano, Lindsay N., Reash, Natalie F., Eagle, Michelle, Lowes, Linda P.

“…Clinical outcome assessments of function or strength, assessed by physical therapists, are commonly used as primary endpoints in clinical trials, natural…”
Get full text

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…”
Get full text

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…”
Get more information

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…”
Get full text

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…”
Get full text

Comparison of strength testing modalities in dysferlinopathy by Reash, Natalie F., James, Meredith K., Alfano, Lindsay N., Mayhew, Anna G., Jacobs, Marni, Iammarino, Megan A., Holsten, Scott, Sakamoto, Chikako, Tateishi, Takayuki, Yajima, Hiroyuki, Duong, Tina, Wolf, Brittney, Gee, Richard, Bharucha‐Goebel, Diana X., Bravver, Elena, Mori‐Yoshimura, Madoka, Bushby, Kate, Rufibach, Laura E., Straub, Volker, Lowes, Linda P.

“…Introduction/Aims Dysferlinopathy demonstrates heterogeneity in muscle weakness between patients, which can progress at different rates over time. Changing…”
Get full text

Validity and Reliability of the Neuromuscular Gross Motor Outcome by Alfano, Lindsay N., Iammarino, Megan A., Reash, Natalie F., Powers, Brenna R., Shannon, Kiana, Connolly, Anne M., Waldrop, Megan A., Noritz, Garey H., Shell, Richard, Tsao, Chang-Yong, Flanigan, Kevin M., Mendell, Jerry R., Lowes, Linda P.

“…Approved treatments in spinal muscular atrophy (SMA) have resulted in unprecedented gains for many individuals. Use of available outcomes, typically developed…”
Get full text

Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study by Nicolau, Stefan, Malhotra, Jyoti, Kaler, Maryann, Magistrado-Coxen, Pamela, Iammarino, Megan A, Reash, Natalie F, Frair, Emma C, Wijeratne, Saranga, Kelly, Benjamin J, White, Peter, Lowes, Linda P, Waldrop, Megan A, Flanigan, Kevin M

“…Single exon duplications account for disease in a minority of Duchenne muscular dystrophy patients. Exon skipping in these patients has the potential to be…”
Get more information

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

Get full text

Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies by James, Meredith K, Alfano, Lindsay N, Muni-Lofra, Robert, Reash, Natalie F, Sodhi, Jassi, Iammarino, Megan A, Moat, Dionne, Shannon, Kianna, McCallum, Michelle, Richardson, Mark, Eagle, Michelle, Straub, Volker, Marini-Bettolo, Chiara, Lowes, Linda P, Mayhew, Anna G

“…Abstract Objective The North Star Assessment for limb-girdle type muscular dystrophies (NSAD), a clinician-reported outcome measure (ClinRO) of motor…”
Get full text

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…”
Get full text

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…”
Get full text

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…”
Get full text