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Validity and Reliability of Clinical and Patient-Reported Outcomes in Multisystem Proteinopathy 1 Alfano, Lindsay N.; Iammarino, Megan A.; Reash, Natalie F.; Lowes, Linda P.; Pietruszewski, Lindsay; Adderley, Kathleen; Humphrey, Lauren; Knight, Audrey B.; Steiner, Christopher L.; Smith, Melissa A.; Sahenk, Zarife; Connolly, Anne M.; Almomen, Momen; D'Ambrosio, Eleonora S.; Peck, Nathan; Peck, Allison Share Save
Gross motor delays in infants and young boys with Duchenne muscular dystrophy Lowes, Linda P.; Reash, Natalie F.; Iammarino, Megan A.; Connolly, Anne M.; Pietruszewski, Lindsay; Smith, Melissa A.; Peng, Jing; Steiner, Christopher L.; Tsao, Chang-Yong; Waldrop, Megan A.; Flanigan, Kevin M.; Chagat, Shannon; Meyer, Alayne P.; Mendell, Jerry R.; Alfano, Lindsay N. Share Save
Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy Zaidman, Craig M.; Goedeker, Natalie L.; Aqul, Amal A.; Butterfield, Russell J.; Connolly, Anne M.; Crystal, Ronald G.; Godwin, Kara E.; Hor, Kan N.; Mathews, Katherine D.; Proud, Crystal M.; Smyth, Elizabeth Kula; Veerapandiyan, Aravindhan; Watkins, Paul B.; Mendell, Jerry R. Share Save
SNUPN deficiency causes a recessive muscular dystrophy due to RNA mis-splicing and ECM dysregulation Nashabat, Marwan; Nabavizadeh, Nasrinsadat; Saracoglu, Hilal Piril; Saribas, Burak; Avci, Sahin; Borklu, Esra; Beillard, Emmanuel; Yilmaz, Elanur; Uygur, Seyide Ecesu; Kayhan, Cavit Kerem; Bosco, Luca; Eren, Zeynep Bengi; Steindl, Katharina; Richter, Manuela Friederike; Bademci, Guney; Rauch, Anita; Fattahi, Zohreh; Valentino, Maria Lucia; Connolly, Anne M.; Bahr, Angela; Viola, Laura; Bergmann, Anke Katharina; Rocha, Maria Eugenia; Peart, Leshon; Castro-Rojas, Derly Liseth; Bueltmann, Eva; Khan, Suliman; Giarrana, Miriam Liliana; Teleanu, Raluca Ioana; Gonzalez, Joanna Michelle; Pini, Antonella; Schadlich, Ines Sophie; Vill, Katharina; Brugger, Melanie; Zuchner, Stephan; Pinto, Andreia; Donkervoort, Sandra; Bivona, Stephanie Ann; Riza, Anca; Streata, Ioana; Glaeser, Dieter; Baquero-Montoya, Carolina; Garcia-Restrepo, Natalia; Kotzaeridou, Urania; Brunet, Theresa; Epure, Diana Anamaria; Bertoli-Avella, Aida; Kariminejad, Ariana; Tekin, Mustafa; von Hardenberg, Sandra; Boennemann, Carsten G.; Stettner, Georg M.; Zanni, Ginevra; Kayserili, Huelya; Oflazer, Zehra Piraye; Escande-Beillard, Nathalie Share Save
The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report Armstrong, 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 Share Save
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Findings from the Longitudinal CINRG Becker Natural History Study 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. Share Save
Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022 Phung, Kim; Crabtree, Nicola; Connolly, Anne M.; Furlong, Pat; Hoffman, Eric P.; Jackowski, Stefan A.; Jayash, Soher Nagi; Johnson, Alex; Koujok, Khaldoun; Munns, Craig F.; Niks, Erik; Rauch, Frank; Schrader, Rachel; Turner, Cathy; Vroom, Elizabeth; Weber, David R.; Wong, Brenda L.; Guglieri, Michela; Ward, Leanne M.; Wong, Sze Choong Share Save
Pamrevlumab, a Fully Human Monoclonal Antibody Targeting Connective Tissue Growth Factor, for Non-Ambulatory Patients with Duchenne Muscular Dystrophy Connolly, Anne M.; Zaidman, Craig M.; Brandsema, John F.; Phan, Han C.; Tian, Cuixia; Zhang, Xueping; Li, Jack; Eisner, Mark D.; Carrier, Ewa Share Save
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG) 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 Share Save
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Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study Darras, Basil; Mercuri, Eugenio; Strauss, Kevin; Day, John; Chien, Yin-Hsiu; Masson, Ricardo; Wigderson, Melissa; Alecu, Iulian; Ballarini, Nicolas; Mehl, Lesa; Marra, Jonathan; Connolly, Anne Share Save
INEQUALITIES IN CONTRACEPTION USE, CONTRACEPTION SERVICE ACCESS AND PREGNANCY PLANNING DURING THE COVID-19 PANDEMIC IN BRITAIN: NATSAL-COVID WAVE 2 SURVEY Baxter, Andrew; Geary, Rebecca; Dema, Emily; Perez, Raquel Boso; Riddell, Julie; Willis, Malachi; Connolly, Anne; Copas, Andrew; Bonell, Chris; Sonnenberg, Pam; Mercer, Cath; Clifton, Zoazig; Field, Nigel; Mitchell, Kirstin Share Save
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy Waldrop, Megan A.; Moore, Steven A.; Mathews, Katherine D.; Darbro, Benjamin W.; Medne, Livja; Finkel, Richard; Connolly, Anne M.; Crawford, Thomas O.; Drachman, Daniel; Wein, Nicolas; Habib, Ali A.; Krzesniak-Swinarska, Monika A.; Zaidman, Craig M.; Collins, James J.; Jokela, Manu; Udd, Bjarne; Day, John W.; Ortiz-Guerrero, Gloria; Statland, Jeff; Butterfield, Russell J.; Dunn, Diane M.; Weiss, Robert B.; Flanigan, Kevin M. Share Save
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial McDonald, Craig M.; Shieh, Perry B.; Abdel-Hamid, Hoda Z.; Connolly, Anne M.; Ciafaloni, Emma; Wagner, Kathryn R.; Goemans, Nathalie; Mercuri, Eugenio; Khan, Navid; Koenig, Erica; Malhotra, Jyoti; Zhang, Wenfei; Han, Baoguang; Mendell, Jerry R. Share Save
Validity and Reliability of the Neuromuscular Gross Motor Outcome 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. Share Save
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study (vol 18, pg 1127, 2021) Rudnicki, 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. Share Save
Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis Mohassel, Payam; Donkervoort, Sandra; Lone, Museer A.; Nalls, Matthew; Gable, Kenneth; Gupta, Sita D.; Foley, A. Reghan; Hu, Ying; Saute, Jonas Alex Morales; Moreira, Ana Lucila; Kok, Fernando; Introna, Alessandro; Logroscino, Giancarlo; Grunseich, Christopher; Nickolls, Alec R.; Pourshafie, Naemeh; Neuhaus, Sarah B.; Saade, Dimah; Gangfuss, Andrea; Koelbel, Heike; Piccus, Zoe; Le Pichon, Claire E.; Fiorillo, Chiara; Ly, Cindy V.; Topf, Ana; Brady, Lauren; Specht, Sabine; Zidell, Aliza; Pedro, Helio; Mittelmann, Eric; Thomas, Florian P.; Chao, Katherine R.; Konersman, Chamindra G.; Cho, Megan T.; Brandt, Tracy; Straub, Volker; Connolly, Anne M.; Schara, Ulrike; Roos, Andreas; Tarnopolsky, Mark; Hoke, Ahmet; Brown, Robert H.; Lee, Chia-Hsueh; Hornemann, Thorsten; Dunn, Teresa M.; Bonnemann, Carsten G. Share Save
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