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Random forest: random results or meaningful insights for patients with facioscapulohumeral muscular dystrophy?
Published in:
2021
By:
- Alfano, Lindsay N;
- Mozaffar, Tahseen
Publication type:
journal article
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.
Published in:
PLoS ONE, 2024, v. 19, n. 5, p. 1, doi. 10.1371/journal.pone.0300700
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.
Publication type:
Article
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.
Published in:
PLoS ONE, 2024, v. 19, n. 3, p. 1, doi. 10.1371/journal.pone.0300244
By:
- Iammarino, Megan A.;
- Alfano, Lindsay N.;
- Reash, Natalie F.;
- Sabo, Brenna;
- Conroy, Sara;
- Noritz, Garey;
- Wendland, Madalynn;
- Lowes, Linda P.
Publication type:
Article
Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Published in:
Muscle & Nerve, 2024, v. 69, n. 1, p. 93, doi. 10.1002/mus.27955
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.
Publication type:
Article
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy.
Published in:
Pediatric Pulmonology, 2019, v. 54, n. 2, p. 179, doi. 10.1002/ppul.24203
By:
- Al‐Zaidy, Samiah;
- Pickard, A. Simon;
- Kotha, Kavitha;
- Alfano, Lindsay N.;
- Lowes, Linda;
- Paul, Grace;
- Church, Kathleen;
- Lehman, Kelly;
- Sproule, Douglas M.;
- Dabbous, Omar;
- Maru, Benit;
- Berry, Katherine;
- Arnold, W. David;
- Kissel, John T.;
- Mendell, Jerry R.;
- Shell, Richard
Publication type:
Article
Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.
Published in:
Frontiers in Neurology, 2022, v. 13, p. 1, doi. 10.3389/fneur.2022.828525
By:
- Mayhew, Anna G.;
- James, Meredith K.;
- Moore, Ursula;
- Sutherland, Helen;
- Jacobs, Marni;
- Jia Feng;
- Lowes, Linda Pax;
- Alfano, Lindsay N.;
- Lofra, Robert Muni;
- Rufibach, Laura E.;
- Rose, Kristy;
- Duong, Tina;
- Bello, Luca;
- Pedrosa-Hernández, Irene;
- Holsten, Scott;
- Chikako Sakamoto;
- Canal, Aurélie;
- Práxedes, Nieves Sánchez-Aguilera;
- Thiele, Simone;
- Siener, Catherine
Publication type:
Article
Comparison of strength testing modalities in dysferlinopathy.
Published in:
Muscle & Nerve, 2022, v. 66, n. 2, p. 159, doi. 10.1002/mus.27570
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;
- de Wolf, Brittney;
- Gee, Richard;
- Bharucha‐Goebel, Diana X.;
- Bravver, Elena;
- Mori‐Yoshimura, Madoka;
- Bushby, Kate;
- Rufibach, Laura E.;
- Straub, Volker;
- Lowes, Linda P.
Publication type:
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
Modeling functional decline over time in sporadic inclusion body myositis.
Published in:
2017
By:
- Alfano, Lindsay N.;
- Yin, Han;
- Dvorchik, Igor;
- Maus, Elizabeth G.;
- Flanigan, Kevin M.;
- Mendell, Jerry R.;
- Lowes, Linda P.
Publication type:
journal article
Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up.
Published in:
2016
By:
- Connolly, Anne M.;
- Florence, Julaine M.;
- Zaidman, Craig M.;
- Golumbek, Paul T.;
- Mendell, Jerry R.;
- Flanigan, Kevin M.;
- Karachunski, Peter I.;
- Day, John W.;
- McDonald, Craig M.;
- Darras, Basil T.;
- Kang, Peter B.;
- Siener, Catherine A.;
- Gadeken, Rebecca K.;
- Anand, Pallavi;
- Schierbecker, Jeanine R.;
- Malkus, Elizabeth C.;
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Johnson, Linda;
- Nicorici, Alina
Publication type:
journal article
Reliability and validity of active-seated: An outcome in dystrophinopathy.
Published in:
Muscle & Nerve, 2015, v. 52, n. 3, p. 356, doi. 10.1002/mus.24557
By:
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Crawfis, Roger;
- Berry, Katherine;
- Yin, Han;
- Dvorchik, Igor;
- Flanigan, Kevin M.;
- Mendell, Jerry R.
Publication type:
Article
Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy.
Published in:
Muscle & Nerve, 2015, v. 51, n. 4, p. 522, doi. 10.1002/mus.24346
By:
- Connolly, Anne M.;
- Malkus, Elizabeth C.;
- Mendell, Jerry R.;
- Flanigan, Kevin M.;
- Miller, J. Philip;
- Schierbecker, Jeanine R.;
- Siener, Catherine A.;
- Golumbek, Paul T.;
- Zaidman, Craig M.;
- Mcdonald, Craig M.;
- Johnson, Linda;
- Nicorici, Alina;
- Karachunski, Peter I.;
- Day, John W.;
- Kelecic, Jason M.;
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Darras, Basil T.;
- Kang, Peter B.;
- Quigley, Janet
Publication type:
Article
Correlation of knee strength to functional outcomes in becker muscular dystrophy.
Published in:
Muscle & Nerve, 2013, v. 47, n. 4, p. 550, doi. 10.1002/mus.23660
By:
- Alfano, Lindsay N.;
- Lowes, Linda P.;
- Flanigan, Kevin M.;
- Mendell, Jerry R.
Publication type:
Article
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.
Published in:
Molecular Therapy, 2015, v. 23, n. 1, p. 192, doi. 10.1038/mt.2014.200
By:
- Mendell, Jerry R;
- Sahenk, Zarife;
- Malik, Vinod;
- Gomez, Ana M;
- Flanigan, Kevin M;
- Lowes, Linda P;
- Alfano, Lindsay N;
- Berry, Katherine;
- Meadows, Eric;
- Lewis, Sarah;
- Braun, Lyndsey;
- Shontz, Kim;
- Rouhana, Maria;
- Clark, Kelly Reed;
- Rosales, Xiomara Q;
- Al-Zaidy, Samiah;
- Govoni, Alessandra;
- Rodino-Klapac, Louise R;
- Hogan, Mark J;
- Kaspar, Brian K
Publication type:
Article
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
Published in:
JAMA Neurology, 2021, v. 78, n. 7, p. 834, doi. 10.1001/jamaneurol.2021.1272
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
Publication type:
Article
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Published in:
JAMA Neurology, 2020, v. 77, n. 9, p. 1122, doi. 10.1001/jamaneurol.2020.1484
By:
- Mendell, Jerry R.;
- Sahenk, Zarife;
- Lehman, Kelly;
- Nease, Carrie;
- Lowes, Linda P.;
- Miller, Natalie F.;
- Iammarino, Megan A.;
- Alfano, Lindsay N.;
- Nicholl, Amanda;
- Al-Zaidy, Samiah;
- Lewis, Sarah;
- Church, Kathleen;
- Shell, Richard;
- Cripe, Linda H.;
- Potter, Rachael A.;
- Griffin, Danielle A.;
- Pozsgai, Eric;
- Dugar, Ashish;
- Hogan, Mark;
- Rodino-Klapac, Louise R.
Publication type:
Article
Proof of Concept of the Ability of the Kinect to Quantify Upper Extremity Function in Dystrophinopathy.
Published in:
PLoS Currents, 2013, p. 566, doi. 10.1371/currents.md.9ab5d872bbb944c6035c9f9bfd314ee2
By:
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Yetter, Brent A.;
- Worthen-Chaudhari, Lise;
- Hinchman, William;
- Savage, Jordan;
- Samona, Patrick;
- Flanigan, Kevin M.;
- Mendell, Jerry R.
Publication type:
Article
Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy.
Published in:
2022
By:
- Korb, Manisha;
- Peck, Allison;
- Alfano, Lindsay N.;
- Berger, Kenneth I.;
- James, Meredith K.;
- Ghoshal, Nupur;
- Healzer, Elise;
- Henchcliffe, Claire;
- Khan, Shaida;
- Mammen, Pradeep P. A.;
- Patel, Sujata;
- Pfeffer, Gerald;
- Ralston, Stuart H.;
- Roy, Bhaskar;
- Seeley, William W.;
- Swenson, Andrea;
- Mozaffar, Tahseen;
- Weihl, Conrad;
- Kimonis, Virginia;
- the VCP Standards of Care Working Group
Publication type:
journal 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
Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 4, p. 469, doi. 10.3233/JND-200548
By:
- Mendell, Jerry R.;
- Khan, Navid;
- Sha, Nanshi;
- Eliopoulos, Helen;
- McDonald, Craig M.;
- Goemans, Nathalie;
- Mercuri, Eugenio;
- Lowes, Linda P.;
- Alfano, Lindsay N.
Publication type:
Article
WiTNNess: An international natural history study of infantile‐onset TNNT1 myopathy.
Published in:
Annals of Clinical & Translational Neurology, 2023, v. 10, n. 11, p. 1972, doi. 10.1002/acn3.51884
By:
- Strauss, Kevin A.;
- Carson, Vincent J.;
- Bolettieri, Emilienne;
- Everett, Mariah;
- Bollinger, Ashton;
- Bowser, Lauren E.;
- Beiler, Keturah;
- Young, Millie;
- Edvardson, Simon;
- Fraenkel, Nitay;
- D'Amico, Adele;
- Bertini, Enrico;
- Lingappa, Lokesh;
- Chowdhury, Devyani;
- Lowes, Linda P.;
- Iammarino, Megan;
- Alfano, Lindsay N.;
- Brigatti, Karlla W.
Publication type:
Article
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.
Published in:
2021
By:
- Jacobs, Marni B.;
- James, Meredoith K.;
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Eagle, Michelle;
- Muni Lofra, Robert;
- Moore, Ursula;
- Feng, Jia;
- Rufibach, Laura E.;
- Rose, Kristy;
- Duong, Tina;
- Bello, Luca;
- Pedrosa‐Hernández, Irene;
- Holsten, Scott;
- Sakamoto, Chikako;
- Canal, Aurélie;
- Sanchez‐Aguilera Práxedes, Nieves;
- Thiele, Simone;
- Siener, Catherine;
- Vandevelde, Bruno
Publication type:
journal article
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
Published in:
2016
By:
- Mendell, Jerry R.;
- Goemans, Nathalie;
- Lowes, Linda P.;
- Alfano, Lindsay N.;
- Berry, Katherine;
- Shao, James;
- Kaye, Edward M.;
- Mercuri, Eugenio;
- Hamid, Hoda Abdel;
- Byrne, Barry J.;
- Connolly, Anne M.;
- Dracker, Robert A.;
- Matthew Frank, L.;
- Heydemann, Peter T.;
- O'Brien, Kevin C.;
- Sparks, Susan E.;
- Specht, Linda A.;
- Rodino‐Klapac, Louise;
- Sahenk, Zarife;
- Al‐Zaidy, Samiah
Publication type:
journal article
ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy.
Published in:
2020
By:
- Alfano, Lindsay N;
- Miller, Natalie F;
- Iammarino, Megan A;
- Moore Clingenpeel, Melissa;
- Lowes, Suzanne L;
- Dugan, Margaret E;
- Kissel, John T;
- Al Zaidy, Samiah;
- Tsao, Chang‐Yong;
- Lowes, Linda P;
- Tsao, Chang-Yong
Publication type:
journal article
Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases.
Published in:
Frontiers in Genetics, 2021, v. 12, p. 1, doi. 10.3389/fgene.2021.735936
By:
- Duong, Tina;
- Krosschell, Kristin J.;
- James, Meredith K.;
- Nelson, Leslie;
- Alfano, Lindsay N.;
- Eichinger, Katy;
- Mazzone, Elena;
- Rose, Kristy;
- Lowes, Linda P.;
- Mayhew, Anna;
- Florence, Julaine;
- King, Wendy;
- Senesac, Claudia R.;
- Eagle, Michelle
Publication type:
Article
Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic.
Published in:
Frontiers in Genetics, 2021, v. 12, p. 1, doi. 10.3389/fgene.2021.735538
By:
- James, Meredith K.;
- Rose, Kristy;
- Alfano, Lindsay N.;
- Reash, Natalie F.;
- Eagle, Michelle;
- Lowes, Linda P.
Publication type:
Article
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy.
Published in:
World Journal of Pediatrics, 2019, v. 15, n. 3, p. 219, doi. 10.1007/s12519-019-00242-6
By:
- Ke, Qing;
- Zhao, Zheng-Yan;
- Mendell, Jerry R.;
- Baker, Mei;
- Wiley, Veronica;
- Kwon, Jennifer M.;
- Alfano, Lindsay N.;
- Connolly, Anne M.;
- Jay, Catherine;
- Polari, Hanna;
- Ciafaloni, Emma;
- Qi, Ming;
- Griggs, Robert C.;
- Gatheridge, Michele A.
Publication type:
Article
Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy.
Published in:
PLoS ONE, 2022, v. 17, n. 4, p. 1, doi. 10.1371/journal.pone.0266845
By:
- Contesse, Marielle G.;
- Lowes, Linda P.;
- White, Michelle K.;
- Dalle Pazze, Laura;
- McSherry, Christine;
- Alfano, Lindsay N.;
- Iammarino, Megan;
- Reash, Natalie;
- Bonarrigo, Kelly;
- Kiefer, Michael;
- Laubscher, Katie;
- McIntyre, Melissa;
- Mockler, Shelley;
- Nelson, Leslie;
- Vogel, Leslie;
- Leffler, Mindy G.
Publication type:
Article
Emerging therapeutic options for sporadic inclusion body myositis.
Published in:
2015
By:
- Alfano, Lindsay N.;
- Lowes, Linda P.
Publication type:
journal article

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