Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease von Rao, Vamshi K., Kapp, Daniel, Schroth, Mary

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Palliative care in Duchenne muscular dystrophy: Goals of care discussions and beyond von Veerapandiyan, Aravindhan, Rao, Vamshi K.

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Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial von Clemens, Paula R, Rao, Vamshi K, Connolly, Anne M, Harper, Amy D, Mah, Jean K, Smith, Edward C, McDonald, Craig M, Zaidman, Craig M, Morgenroth, Lauren P, Osaki, Hironori, Satou, Youhei, Yamashita, Taishi, Hoffman, Eric P

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Combination molecular therapies for type 1 spinal muscular atrophy von Harada, Yohei, Rao, Vamshi K., Arya, Kapil, Kuntz, Nancy L., DiDonato, Christine J., Napchan‐Pomerantz, Galia, Agarwal, Amit, Stefans, Vikki, Katsuno, Masahisa, Veerapandiyan, Aravindhan

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Prevalence of Attention-Deficit/Hyperactivity Disorder and Autism Spectrum Disorder in Individuals With Dystrophinopathy at a Tertiary Care Center in Chicago von Diehl, Emily, O'Neill, Meghan, Gray, Larry, Schwaede, Abigail, Kuntz, Nancy, Rao, Vamshi K.

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Predicting outcomes for spinal muscular atrophy: When the diagnosis no longer means what it used to mean von Toaz, Erin E., Pinto, Nisha M., Rao, Vamshi K., Cheon, Eric C.

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Measuring the Efficacy of Thymectomy for Pediatric Myasthenia Gravis Across Tertiary Children's Hospitals von De Boer, Christopher, Zeineddin, Suhail, Ott, Katherine, Hu, Andrew, Linton, Samuel, George, Michael, Rao, Vamshi K., Abdullah, Fizan, Chin, Anthony, Goldstein, Seth D.

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The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic von Veerapandiyan, Aravindhan, Wagner, Kathryn R., Apkon, Susan, McDonald, Craig M., Mathews, Katherine D., Parsons, Julie A., Wong, Brenda L., Eichinger, Katy, Shieh, Perry B., Butterfield, Russell J., Rao, Vamshi K., Smith, Edward C., Proud, Crystal M., Connolly, Anne M., Ciafaloni, Emma

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Expansion of the phenotypic spectrum associated with pathogenic missense variation in DHX16 von Drackley, Andy, De Simone, Lenika, Kuntz, Nancy, Rahmani, Safa, Ing, Alexander, Rao, Vamshi K., Rathbun, Pamela, Yap, Kai Lee

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Axonal polyneuropathy and ataxia in children: consider Perrault Syndrome, a case report von Munson, Hannah E, De Simone, Lenika, Schwaede, Abigail, Bhatia, Avanti, Mithal, Divakar S, Young, Nancy, Kuntz, Nancy, Rao, Vamshi K

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Spinal muscular atrophy care in the COVID‐19 pandemic era von Veerapandiyan, Aravindhan, Connolly, Anne M., Finkel, Richard S., Arya, Kapil, Mathews, Katherine D., Smith, Edward C., Castro, Diana, Butterfield, Russell J., Parsons, Julie A., Servais, Laurent, Kuntz, Nancy, Rao, Vamshi K., Brandsema, John F., Mercuri, Eugenio, Ciafaloni, Emma

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Eculizumab in Adolescent Patients With Refractory Generalized Myasthenia Gravis: A Phase 3, Open-Label, Multicenter Study von Brandsema, John F., Ginsberg, Matthew, Hoshino, Hideki, Mimaki, Masakazu, Nagata, Satoru, Rao, Vamshi K., Ruzhansky, Katherine, Suresh, Niraja, Tiongson, Emmanuelle, Yamanouchi, Hideo, Frick, Glen, Hicks, Eden, Liao, Serena, Howard, James F.

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EPG5-related Vici syndrome: a paradigm of neurodevelopmental disorders with defective autophagy von Byrne, Susan, Jansen, Lara, U-King-Im, Jean-Marie, Siddiqui, Ata, Lidov, Hart G W, Bodi, Istvan, Smith, Luke, Mein, Rachael, Cullup, Thomas, Dionisi-Vici, Carlo, Al-Gazali, Lihadh, Al-Owain, Mohammed, Bruwer, Zandre, Al Thihli, Khalid, El-Garhy, Rana, Flanigan, Kevin M, Manickam, Kandamurugu, Zmuda, Erik, Banks, Wesley, Gershoni-Baruch, Ruth, Mandel, Hanna, Dagan, Efrat, Raas-Rothschild, Annick, Barash, Hila, Filloux, Francis, Creel, Donnell, Harris, Michael, Hamosh, Ada, Kölker, Stefan, Ebrahimi-Fakhari, Darius, Hoffmann, Georg F, Manchester, David, Boyer, Philip J, Manzur, Adnan Y, Lourenco, Charles Marques, Pilz, Daniela T, Kamath, Arveen, Prabhakar, Prab, Rao, Vamshi K, Rogers, R Curtis, Ryan, Monique M, Brown, Natasha J, McLean, Catriona A, Said, Edith, Schara, Ulrike, Stein, Anja, Sewry, Caroline, Travan, Laura, Wijburg, Frits A, Zenker, Martin, Mohammed, Shehla, Fanto, Manolis, Gautel, Mathias, Jungbluth, Heinz

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Utility of Repetitive Nerve Stimulation in Myopathies von Schwaede, Abigail, Buehner, Amber N, Rao, Vamshi K

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Muscle channelopathies: A review von McGowan, Bridget R., Schwaede, Abigail N., De Simone, Lenika, Rao, Vamshi K.

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A case report of riboflavin transporter deficiency: A novel heterozygous pathogenic variant in the SLC52A3 gene von Tranel, Elizabeth S., McGowan, Bridget, Drackley, Andy, Epstein, Leon G., Rao, Vamshi K., Kuntz, Nancy L., Schwaede, Abigail N.

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An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy von Kwon, 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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Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy von Clemens, Paula R., Rao, Vamshi K., Connolly, Anne M., Harper, Amy D., Mah, Jean K., McDonald, Craig M., Smith, Edward C., Zaidman, Craig M., Nakagawa, Tomoyuki, Hoffman, Eric P.

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Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial von Dang, 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, Haberlová, 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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PIGQ glycosylphosphatidylinositol‐anchored protein deficiency: Characterizing the phenotype von Starr, Lois J., Spranger, Jürgen W., Rao, Vamshi K., Lutz, Richard, Yetman, Anji T.

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