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Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5‐year update of the NURTURE study.
Published in:
Muscle & Nerve, 2023, v. 68, n. 2, p. 157, doi. 10.1002/mus.27853
By:
- Crawford, Thomas O.;
- Swoboda, Kathryn J.;
- De Vivo, Darryl C.;
- Bertini, Enrico;
- Hwu, Wuh‐Liang;
- Finkel, Richard S.;
- Kirschner, Janbernd;
- Kuntz, Nancy L.;
- Nazario, Aledie Navas;
- Parsons, Julie A.;
- Pechmann, Astrid;
- Ryan, Monique M.;
- Butterfield, Russell J.;
- Topaloglu, Haluk;
- Ben‐Omran, Tawfeg;
- Sansone, Valeria A.;
- Jong, Yuh‐Jyh;
- Shu, Francy;
- Zhu, Cong;
- Raynaud, Stephanie
Publication type:
Article
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies.
Published in:
Journal of Multidisciplinary Healthcare, 2024, v. 17, p. 4199, doi. 10.2147/JMDH.S458181
By:
- Fischer, Ryan;
- Furlong, Pat;
- Kennedy, Annie;
- Maynard, Kelly;
- Penrod, Marissa;
- Miller, Debra;
- Laverty, Chamindra G;
- Lowes, Linda P;
- Kuntz, Nancy L;
- Shieh, Perry B;
- Kondejewski, Jane;
- Neumann, Peter J;
- Shafrin, Jason;
- Willke, Richard J
Publication type:
Article
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial.
Published in:
Muscle & Nerve, 2021, v. 64, n. 3, p. 285, doi. 10.1002/mus.27347
By:
- Wagner, Kathryn R.;
- Kuntz, Nancy L.;
- Koenig, Erica;
- East, Lilly;
- Upadhyay, Sameer;
- Han, Baoguang;
- Shieh, Perry B.
Publication type:
Article
Combination molecular therapies for type 1 spinal muscular atrophy.
Published in:
2020
By:
- Harada, Yohei;
- Rao, Vamshi K.;
- Arya, Kapil;
- Kuntz, Nancy L.;
- DiDonato, Christine J.;
- Napchan‐Pomerantz, Galia;
- Agarwal, Amit;
- Stefans, Vikki;
- Katsuno, Masahisa;
- Veerapandiyan, Aravindhan;
- Napchan-Pomerantz, Galia
Publication type:
journal article
Utility and practice of electrodiagnostic testing in the pediatric population: An AANEM consensus statement.
Published in:
2020
By:
- Kang, Peter B.;
- McMillan, Hugh J.;
- Kuntz, Nancy L.;
- Lehky, Tanya J.;
- Alter, Katharine E.;
- Fitzpatrick, Kevin F.;
- El Kosseifi, Charbel;
- Quijano‐Roy, Susana;
- Quijano-Roy, Susana;
- Professional Practice Committee of the American Association of Neuromuscular & Electrodiagnostic Medicine
Publication type:
journal article
Twice‐weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
Published in:
Muscle & Nerve, 2019, v. 59, n. 6, p. 650, doi. 10.1002/mus.26441
By:
- Connolly, Anne M.;
- Zaidman, Craig M.;
- Golumbek, Paul T.;
- Cradock, Mary M.;
- Flanigan, Kevin M.;
- Kuntz, Nancy L.;
- Finkel, Richard S.;
- McDonald, Craig M.;
- Iannaccone, Susan T.;
- Anand, Pallavi;
- Siener, Catherine A.;
- Florence, Julaine M.;
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Johnson, Linda B.;
- Nicorici, Alina;
- Nelson, Leslie L.;
- Mendell, Jerry R.
Publication type:
Article
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
Published in:
2019
By:
- Connolly, Anne M;
- Zaidman, Craig M;
- Golumbek, Paul T;
- Cradock, Mary M;
- Flanigan, Kevin M;
- Kuntz, Nancy L;
- Finkel, Richard S;
- McDonald, Craig M;
- Iannaccone, Susan T;
- Anand, Pallavi;
- Siener, Catherine A;
- Florence, Julaine M;
- Lowes, Linda P;
- Alfano, Lindsay N;
- Johnson, Linda B;
- Nicorici, Alina;
- Nelson, Leslie L;
- Mendell, Jerry R;
- MDA DMD Clinical Research Network
Publication type:
journal article
A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.
Published in:
2018
By:
- Beggs, Alan H.;
- Byrne, Barry J.;
- De Chastonay, Sabine;
- Haselkorn, Tmirah;
- Hughes, Imelda;
- James, Emma S.;
- Kuntz, Nancy L.;
- Simon, Jennifer;
- Swanson, Lindsay C.;
- Yang, Michele L.;
- Yu, Zi‐Fan;
- Yum, Sabrina W.;
- Prasad, Suyash;
- Yu, Zi-Fan
Publication type:
journal article
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Published in:
JAMA Network Open, 2022, v. 5, n. 1, p. e2144178, doi. 10.1001/jamanetworkopen.2021.44178
By:
- Mah, Jean K.;
- Clemens, Paula R.;
- Guglieri, Michela;
- Smith, Edward C.;
- Finkel, Richard S.;
- Tulinius, Mar;
- Nevo, Yoram;
- Ryan, Monique M.;
- Webster, Richard;
- Castro, Diana;
- Kuntz, Nancy L.;
- McDonald, Craig M.;
- Damsker, Jesse M.;
- Schwartz, Benjamin D.;
- Mengle-Gaw, Laurel J.;
- Jackowski, Stefan;
- Stimpson, Georgia;
- Ridout, Deborah A.;
- Ayyar-Gupta, Vandana;
- Baranello, Giovanni
Publication type:
Article
Use of the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) in Spinal Muscular Atrophy.
Published in:
Journal of Clinical Medicine, 2024, v. 13, n. 4, p. 921, doi. 10.3390/jcm13040921
By:
- Brown, 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.
Publication type:
Article
Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model.
Published in:
CNS Drugs, 2022, v. 36, n. 2, p. 181, doi. 10.1007/s40263-022-00899-0
By:
- MacCannell, Drew;
- Berger, Zdenek;
- Kirschner, Janbernd;
- Mercuri, Eugenio;
- Farrar, Michelle A.;
- Iannaccone, Susan T.;
- Kuntz, Nancy L.;
- Finkel, Richard S.;
- Valente, Marta;
- Muntoni, Francesco
Publication type:
Article
Medical management of muscle weakness in Duchenne muscular dystrophy.
Published in:
PLoS ONE, 2020, v. 15, n. 10, p. 1, doi. 10.1371/journal.pone.0240687
By:
- Rivera, Sarah R.;
- Jhamb, Sumit K.;
- Abdel-Hamid, Hoda Z.;
- Acsadi, Gyula;
- Brandsema, John;
- Ciafaloni, Emma;
- Darras, Basil T.;
- Iannaccone, Susan T.;
- Konersman, Chamindra G.;
- Kuntz, Nancy L.;
- McDonald, Craig M.;
- Parsons, Julie A.;
- Tesi Rocha, Carolina;
- Zaidman, Craig M.;
- Butterfield, Russell J.;
- Connolly, Anne M.;
- Mathews, Katherine D.
Publication type:
Article
Longitudinal study of intraneural perineurioma--a benign, focal hypertrophic neuropathy of youth.
Published in:
Brain: A Journal of Neurology, 2009, v. 132, n. 8, p. 2265, doi. 10.1093/brain/awp169
By:
- Mauermann, Michelle L.;
- Amrami, Kimberly K.;
- Kuntz, Nancy L.;
- Spinner, Robert J.;
- Dyck, Peter J.;
- Bosch, E. Peter;
- Engelstad, JaNean;
- Felmlee, Joel P.;
- Dyck, P. James B.
Publication type:
Article
Findings from the Longitudinal CINRG Becker Natural History Study.
Published in:
Journal of Neuromuscular Diseases, 2024, v. 11, n. 1, p. 201, doi. 10.3233/JND-230178
By:
- Clemens, 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.
Publication type:
Article
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).
Published in:
Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 389, doi. 10.3233/JND-221560
By:
- Finkel, 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
Publication type:
Article
Longitudinal Assessment of Timed Function Tests in Ambulatory Individuals with SMA Treated with Nusinersen.
Published in:
Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 337, doi. 10.3233/JND-221519
By:
- Krosschell, 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.
Publication type:
Article
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Published in:
Journal of Neuromuscular Diseases, 2022, v. 9, n. 4, p. 503, doi. 10.3233/JND-210781
By:
- Dowling, James J.;
- Müller-Felber, Wolfgang;
- Smith, Barbara K.;
- Bönnemann, Carsten G.;
- Kuntz, Nancy L.;
- Muntoni, Francesco;
- Servais, Laurent;
- Alfano, Lindsay N.;
- Beggs, Alan H.;
- Bilder, Deborah A.;
- Blaschek, Astrid;
- Duong, Tina;
- Graham, Robert J.;
- Jain, Minal;
- Lawlor, Michael W.;
- Lee, Jun;
- Coats, Julie;
- Lilien, Charlotte;
- Lowes, Linda P.;
- MacBean, Victoria
Publication type:
Article
Localized Aquadynia Responsive to Clonidine in a 13-Year-Old Girl.
Published in:
Pediatric Dermatology, 2010, v. 27, n. 6, p. 646, doi. 10.1111/j.1525-1470.2010.01132.x
By:
- Lehman, Julia S.;
- Kuntz, Nancy L.;
- Davis, Dawn M. R.
Publication type:
Article
Updates in the Use of Vamorolone and Steroids in the Treatment of Duchenne Muscular Dystrophy.
Published in:
touchREVIEWS in Neurology, 2023, v. 19, n. 2, p. 1, doi. 10.17925/USN.2023.19.2.5
By:
- McGowan, Bridget;
- Kuntz, Nancy L
Publication type:
Article
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
Published in:
2020
By:
- Smith, Edward C.;
- Conklin, Laurie S.;
- Hoffman, Eric P.;
- Clemens, Paula R.;
- Mah, Jean K.;
- Finkel, Richard S.;
- Guglieri, Michela;
- Tulinius, Mar;
- Nevo, Yoram;
- Ryan, Monique M.;
- Webster, Richard;
- Castro, Diana;
- Kuntz, Nancy L.;
- Kerchner, Laurie;
- Morgenroth, Lauren P.;
- Arrieta, Adrienne;
- Shimony, Maya;
- Jaros, Mark;
- Shale, Phil;
- Gordish-Dressman, Heather
Publication type:
journal article
Unusual Case of Relapsing and Remitting Rash With Progressive Motor Sensory Neuropathy and Spinal Cord Atrophy.
Published in:
2012
By:
- Blackburn, Joanna S.;
- Kuntz, Nancy L.;
- Grimason, Michele;
- Wainwright, Mark S.
Publication type:
Case Study
Management of Pediatric Central Nervous System Demyelinating Disorders: Consensus of United States Neurologists.
Published in:
Journal of Child Neurology, 2011, v. 26, n. 6, p. 675, doi. 10.1177/0883073810395141
By:
- Waldman, Amy T.;
- Gorman, Mark P.;
- Rensel, Mary R.;
- Austin, Tracy E.;
- Hertz, Deborah P.;
- Kuntz, Nancy L.
Publication type:
Article
Orthostatic Heart Rate and Blood Pressure in Adolescents: Reference Ranges.
Published in:
Journal of Child Neurology, 2010, v. 25, n. 10, p. 1210, doi. 10.1177/0883073809359539
By:
- Skinner, Joline E.;
- Driscoll, Sherilyn W.;
- Porter, Co-burn J.;
- Brands, Chad K.;
- Pianosi, Paolo T.;
- Kuntz, Nancy L.;
- Nelson, Dawn E.;
- Burkhardt, Barbara E.;
- Bryant, Sandra C.;
- Fischer, Philip R.
Publication type:
Article
Review Article: Nerve Conduction Studies in Infants and Children.
Published in:
Journal of Child Neurology, 1986, v. 1, n. 1, p. 19, doi. 10.1177/088307388600100103
By:
- Miller, Robert G.;
- Kuntz, Nancy L.
Publication type:
Article
Moderate exercise improves function and increases adiponectin in the mdx mouse model of muscular dystrophy.
Published in:
Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-42203-z
By:
- Zelikovich, Aaron S.;
- Quattrocelli, Mattia;
- Salamone, Isabella M.;
- Kuntz, Nancy L.;
- McNally, Elizabeth M.
Publication type:
Article
The electrophysiologic profile of Dejerine-Sottas disease (HMSN III).
Published in:
Muscle & Nerve, 1990, v. 13, n. 7, p. 586, doi. 10.1002/mus.880130705
By:
- Benstead, Timothy J.;
- Kuntz, Nancy L.;
- Miller, Robert G.;
- Daube, Jasper R.
Publication type:
Article

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