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Nancy L. Kuntz

Alberta Children's Hospital

49H指数
204论文数
1.3W被引数
收录论文 56
发表时间
New SMA era: A broad-range tiered assessment of function for the evolving SMA phenotype (EVOLVE-SMA)新SMA时代:针对不断演变的SMA表型的广谱分级功能评估(EVOLVE-SMA)
err2025-12-30
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PREAI
errMeghan Moore Burk; Thomas O Crawford; Kristen Johnson; Susan Apkon; Laurey Brown; Terri Carry; Katie Hoffman; Carolyn Kelley; Nancy L Kuntz; Julie Miller; Jessica Nance; Ruchee Patel; Vamshi Rao; Carly Rothman; Abigail Schwaede; Anne Stratton; Sara Thinger; Jessica Trenkle; Madeline Van Os; Kerry Vela; Tina Duong; Kristin J Krosschell
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Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy开放标签IV期临床试验,评估在脊髓性肌萎缩症患儿中,在onasemnogene abeparvovec治疗后使用nusinersen的效果。
err2025-11-17
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errOAAI
errProud, Crystal M.; Finkel, Richard S.; Parsons, Julie A.; Masson, Riccardo; Brandsema, John F.; Kuntz, Nancy L.; Foster, Richard; Li, Wenjing; Littauer, Ross; Sohn, Jihee; Fradette, Stephanie; Youn, Bora; Paradis, Angela D.
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A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophySAPPHIRE临床试验的简明语言摘要,该试验研究apitegromab在儿童和年轻成人脊髓性肌萎缩症患者中的应用。
err2025-11-13
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errOAAI
errThomas O. Crawford; Laurent Servais; Eugenio Mercuri; Heike Kölbel; Nancy Kuntz; Richard S. Finkel; Jena Krueger; Kaitlin Batley; Sally Dunaway Young; Jing L. Marantz; Guochen Song; Bert Yao; Guolin Zhao; Jose Rossello; Giridhar S. Tirucherai; Elena Stacy Mazzone; Russell J. Butterfield; Marta Gomez Garcia de la Banda; Andreea M. Seferian; Valeria A. Sansone; Liesbeth De Waele; W. Ludo van der Pol; Claude Cances; Astrid Pechmann; Basil T. Darras
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Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4-to <7-year childrenvamorolone(一种解离性皮质类固醇抗炎药)对4至<7岁儿童血清生物标志物的暴露-反应关系
err2025-11-01
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errOAAI
errMummidivarpu, Swati; Dang, Utkarsh J.; Ziemba, Michael; Hathout, Yetrib; Clemens, Paula R.; Damsker, Jesse; Hagerty, Laura; Jusko, William J.; Smith, Edward C.; Mah, Jean K.; Guglieri, Michela; Nevo, Yoram; Kuntz, Nancy; Mcdonald, Craig M.; Ryan, Monique M.; Castro, Diana; Finkel, Richard S.; Conklin, Laurie S.; Mccall, John M.; Nagaraju, Kanneboyina; Van Den Anker, John; Hoffman, Eric P.
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Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial阿皮特格罗玛ab在非行走型2型或3型脊髓性肌萎缩症中的安全性与有效性(SAPPHIRE研究):一项III期、双盲、随机、安慰剂对照试验
err2025-08-13
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PREAI
errThomas O Crawford; Laurent Servais; Eugenio Mercuri; Heike Kölbel; Nancy Kuntz; Richard S Finkel; Jena Krueger; Kaitlin Batley; Sally Dunaway Young; Jing L Marantz; Guochen Song; Bert Yao; Guolin Zhao; Jose Rossello; Giridhar S Tirucherai; Elena Stacy Mazzone; Russell J Butterfield; Marta Gomez Garcia de la Banda; Andreea M Seferian; Valeria A Sansone; Liesbeth De Waele; W Ludo van der Pol; Claude Cances; Astrid Pechmann; Basil T Darras
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Spinal Muscular Atrophy Update in Best Practices
err2024-08-01
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errOAAI
errSchroth, Mary; Deans, Jennifer; Arya, Kapil; Castro, Diana; De Vivo, Darryl C.; Gibbons, Melissa A.; Ionita, Cristian; Kuntz, Nancy L.; Lakhotia, Arpita; Knierbein, Erin Neil; Scoto, Mariacristina; Sejersen, Thomas; Servais, Laurent; Tian, Cuixia; Waldrop, Megan A.; Vazquez-Costa, Juan F.
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Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular DystrophyVamorolone在Duchenne肌营养不良症男孩中48周的疗效和安全性
err2024-03-12
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errOAAI
errDang, Utkarsh J.; Damsker, Jesse M.; Guglieri, Michela; Clemens, Paula R.; Perlman, Seth J.; Smith, Edward C.; Horrocks, Iain; Finkel, Richard S.; Mah, Jean K.; Deconinck, Nicolas; Goemans, Nathalie M.; Haberlova, Jana; Straub, Volker; Mengle-Gaw, Laurel; Schwartz, Benjamin D.; Harper, Amy; Shieh, Perry B.; De Waele, Liesbeth; Castro, Diana; Yang, Michele L.; Ryan, Monique M.; McDonald, Craig M.; Tulinius, Mar; Webster, Richard I.; Mcmillan, Hugh J.; Kuntz, Nancy; Rao, Vamshi K.; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M.; Selby, Kathryn A.; Monduy, Migvis; Nevo, Yoram; Vilchez, Juan J.; Nascimento-Osorio, Andres; Niks, Erik H.; De Groot, Imelda J. M.; Katsalouli, Marina; van den Anker, John N.; Ward, Leanne M.; Leinonen, Mika; D'Alessandro, Andrea L.; Hoffman, Eric P.
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Use of the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) in Spinal Muscular Atrophy
err2024-02-06
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errOAAI
errBrown, Laurey; Hoffman, Katie; Corbo-Galli, Chiara; Dong, Siyuan; Zumpf, Katelyn; Weigel, Christa; Blomgren, Colleen; Munson, Hannah; Bidwell, Jessa; Rao, Vamshi; Kuntz, Nancy L.; Schwaede, Abigail; Krosschell, Kristin J.
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The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report
err2024-01-04
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errOAAI
errArmstrong, Niki; Apkon, Susan; Berggren, Kiera N.; Braun, Catherine; Ciafaloni, Emma; Connolly, Anne; Kennedy, Annie; Kuntz, Nancy; Mathews, Katherine; McGuire, Michelle; Parad, Richard; Scavina, Mena; Scharf, Rebecca J.; Waldrop, Megan
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Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
err2024-01-01
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errOAAI
errLawlor, Michael W.; Schoser, Benedikt; Margeta, Marta; Sewry, Caroline A.; Jones, Karra A.; Shieh, Perry B.; Kuntz, Nancy L.; Smith, Barbara K.; Dowling, James J.; Mueller-Felber, Wolfgang; Boennemann, Carsten G.; Seferian, Andreea M.; Blaschek, Astrid; Neuhaus, Sarah; Foley, A. Reghan; Saade, Dimah N.; Tsuchiya, Etsuko; Qasim, Ummulwara R.; Beatka, Margaret; Prom, Mariah J.; Ott, Emily; Danielson, Susan; Krakau, Paul; Kumar, Suresh N.; Meng, Hui; Vanden Avond, Mark; Wells, Clive; Gordish-Dressman, Heather; Beggs, Alan H.; Christensen, Sarah; Conner, Edward; James, Emma S.; Lee, Jun; Sadhu, Chanchal; Miller, Weston; Sepulveda, Bryan; Varfaj, Fatbardha; Prasad, Suyash; Rico, Salvador
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Findings from the Longitudinal CINRG Becker Natural History Study
err2023-11-17
err5
errOAAI
errClemens, Paula R.; Gordish-Dressman, Heather; Niizawa, Gabriela; Gorni, Ksenija; Guglieri, Michela; Connolly, Anne M.; Wicklund, Matthew; Bertorini, Tulio; Mah, Jean; Thangarajh, Mathula; Smith, Edward C.; Kuntz, Nancy L.; McDonald, Craig M.; Henricson, Erik; Upadhyayula, S.; Byrne, Barry; Manousakis, Georgios; Harper, Amy; Iannaccone, Susan; Dang, Utkarsh J.
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Longitudinal Assessment of Timed Function Tests in Ambulatory Individuals with SMA Treated with Nusinersen
err2023-05-02
err1
PREAI
errKrosschell, Kristin J.; Brown, Laurey; Hoffman, Katie; Zumpf, Katelyn B.; Munson, Hannah; Bidwell, Jessa; Schulte, Duncan P.; Schwaede, Abigail N.; Buehner, Amber N.; DiDonato, Christine J.; Kuntz, Nancy L.; Rao, Vamshi K.
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Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)鞘内注射Onasemnogene Abeparvovec用于坐着,非卧床的脊髓性肌萎缩症患者: I期递增剂量研究 (STRONG)
err2023-05-02
err21
errOAAI
errFinkel, Richard S.; Darras, Basil T.; Mendell, Jerry R.; Day, John W.; Kuntz, Nancy L.; Connolly, Anne M.; Zaidman, Craig M.; Crawford, Thomas O.; Butterfield, Russell J.; Shieh, Perry B.; Tennekoon, Gihan; Brandsema, John F.; Iannaccone, Susan T.; Shoffner, John; Kavanagh, Sarah; Macek, Thomas A.; Tauscher-Wisniewski, Sitra
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AMFR dysfunction causes autosomal recessive spastic paraplegia in human that is amenable to statin treatment in a preclinical model
err2023-04-29
err10
errOAAI
errDeng, Ruizhi; Medico-Salsench, Eva; Nikoncuk, Anita; Ramakrishnan, Reshmi; Lanko, Kristina; Kuhn, Nikolas A.; van der Linde, Herma C.; Lor-Zade, Sarah; Albuainain, Fatimah; Shi, Yuwei; Yousefi, Soheil; Capo, Ivan; van den Herik, Evita Medici; van Slegtenhorst, Marjon; van Minkelen, Rick; Geeven, Geert; Mulder, Monique T.; Ruijter, George J. G.; Luetjohann, Dieter; Jacobs, Edwin H.; Houlden, Henry; Pagnamenta, Alistair T.; Metcalfe, Kay; Jackson, Adam; Banka, Siddharth; De Simone, Lenika; Schwaede, Abigail; Kuntz, Nancy; Palculict, Timothy Blake; Abbas, Safdar; Umair, Muhammad; AlMuhaizea, Mohammed; Colak, Dilek; AlQudairy, Hanan; Alsagob, Maysoon; Pereira, Catarina; Trunzo, Roberta; Karageorgou, Vasiliki; Bertoli-Avella, Aida M.; Bauer, Peter; Bouman, Arjan; Hoefsloot, Lies H.; van Ham, Tjakko J.; Issa, Mahmoud; Zaki, Maha S.; Gleeson, Joseph G.; Willemsen, Rob; Kaya, Namik; Arold, Stefan T.; Maroofian, Reza; Sanderson, Leslie E.; Barakat, Tahsin Stefan
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Baseline Characteristics and Interim Safety in RESPOND: A Phase 4 Study in Children with Spinal Muscular Atrophy (SMA) Treated With Nusinersen After Onasemnogene Abeparvovec
err2023-04-25
err1
PREAI
errKuntz, Nancy; Parsons, Julie; Brandsema, John F.; Proud, Crystal; Finkel, Richard; Swoboda, Kathryn; Masson, Ricardo; Liu, Yingying; Makepeace, Corinne; Paradis, Angela; Berger, Zdenek; Somera-Molina, Kathleen
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An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy
err2022-05-14
err25
errOAAI
errKwon, Jennifer M.; Arya, Kapil; Kuntz, Nancy; Phan, Han C.; Sieburg, Cory; Swoboda, Kathryn J.; Veerapandiyan, Aravindhan; Assman, Beverly; Bader-Weder, Silvia; Dickendesher, Travis L.; Hansen, Jennifer; Lin, Helen; Yan, Ying; Rao, Vamshi K.
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ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findings
err2022-03-01
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errOAAI
errShieh, Perry; Kuntz, Nancy; Dowling, James; Mueller-Felber, Wolfgang; Bonnemann, Carsten; Foley, Dimah; Seferian, Andreea; Servais, Laurent; Lawlor, Michael; Noursalehi, Mojtaba; Prasad, Suyash; Rico, Salvador; Miller, Weston
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Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model
err2022-01-26
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errOAAI
errMacCannell, Drew; Berger, Zdenek; Kirschner, Janbernd; Mercuri, Eugenio; Farrar, Michelle A.; Iannaccone, Susan T.; Kuntz, Nancy L.; Finkel, Richard S.; Valente, Marta; Muntoni, Francesco
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Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study (vol 18, pg 1127, 2021)
err2021-07-01
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errOAAI
errRudnicki, Stacy A.; Andrews, Jinsy A.; Duong, Tina; Cockroft, Bettina M.; Malik, Fady I.; Meng, Lisa; Wei, Jenny; Wolff, Andrew A.; Genge, Angela; Johnson, Nicholas E.; Tesi-Rocha, Carolina; Connolly, Anne M.; Darras, Basil T.; Felice, Kevin; Finkel, Richard S.; Shieh, Perry B.; Mah, Jean K.; Statland, Jeffrey; Campbell, Craig; Habib, Ali A.; Kuntz, Nancy L.; Oskoui, Maryam; Day, John W.
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