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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.

Paula R Clemens, Vamshi K Rao, Anne M Connolly, Amy D Harper, Jean K Mah, Edward C Smith, Craig M McDonald, Craig M Zaidman, Lauren P Morgenroth, Hironori Osaki, Youhei Satou, Taishi Yamashita, Eric P Hoffman

Publication

Combination molecular therapies for type 1 spinal muscular atrophy.

Yohei Harada, Vamshi K Rao, Kapil Arya, Nancy L Kuntz, Christine J DiDonato, Galia Napchan-Pomerantz, Amit Agarwal, Vikki Stefans, Masahisa Katsuno, Aravindhan Veerapandiyan

Publication

Spinal muscular atrophy care in the COVID-19 pandemic era.

Aravindhan Veerapandiyan, Anne M Connolly, Richard S Finkel, Kapil Arya, Katherine D Mathews, Edward C Smith, Diana Castro, Russell J Butterfield, Julie A Parsons, Laurent Servais, Nancy Kuntz, Vamshi K Rao, John F Brandsema, Eugenio Mercuri, Emma Ciafaloni

Publication

The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic.

Aravindhan Veerapandiyan, Kathryn R Wagner, Susan Apkon, Craig M McDonald, Katherine D Mathews, Julie A Parsons, Brenda L Wong, Katy Eichinger, Perry B Shieh, Russell J Butterfield, Vamshi K Rao, Edward C Smith, Crystal M Proud, Anne M Connolly, Emma Ciafaloni

Publication

Utility of Repetitive Nerve Stimulation in Myopathies.

Abigail Schwaede, Amber N Buehner, Vamshi K Rao

Publication

Spinal Muscular Atrophy Diagnosed by Newborn Screening.

Matias Lopez-Chacon, Amber N Buehner, Vamshi K Rao

Publication

PIGQ glycosylphosphatidylinositol‐anchored protein deficiency: Characterizing the phenotype

Lois J Starr, Jürgen W Spranger, Vamshi K Rao, Richard Lutz, Anji T Yetman

Publication

Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease.

Vamshi K Rao, Daniel Kapp, Mary Schroth

Publication

Friedreich’s Ataxia: Clinical Presentation of a Compound Heterozygote Child with a Rare Nonsense Mutation and Comparison with Previously Published Cases

Vamshi K Rao, Christine J DiDonato, Paul D Larsen

Publication

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Richard S Finkel, Eugenio Mercuri, Basil T Darras, Anne M Connolly, Nancy L Kuntz, Janbernd Kirschner, Claudia A Chiriboga, Kayoko Saito, Laurent Servais, Eduardo Tizzano, Haluk Topaloglu, Már Tulinius, Jacqueline Montes, Allan M Glanzman, Kathie Bishop, Z John Zhong, Sarah Gheuens, C Frank Bennett, Eugene Schneider, Wildon Farwell, Darryl C De Vivo

Publication

Guidelines for Corticosteroid use in Treatment of DMD

Vamshi K Rao

Publication

EPG5-related Vici syndrome: a paradigm of neurodevelopmental disorders with defective autophagy.

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

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