Orofacial EMG in Congenital Multiple Cranial Neuropathies

por Rao, Vamshi K.
Publicado 2015

Guidelines for Corticosteroid use in Treatment of DMD

por Rao, Vamshi K.
Publicado 2016

Delay in Diagnosis of Duchenne Muscular Dystrophy

por Rao, Vamshi K., Kuntz, Nancy L.
Publicado 2015

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

por Rao, Vamshi K., Kapp, Daniel, Schroth, Mary
Publicado 2018

Utility of Repetitive Nerve Stimulation in Myopathies

por Schwaede, Abigail, Buehner, Amber N., Rao, Vamshi K.
Publicado 2020

Spinal Muscular Atrophy Diagnosed by Newborn Screening

por Lopez-Chacon, Matias, Buehner, Amber N., Rao, Vamshi K.
Publicado 2019

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

por Rao, Vamshi K., DiDonato, Christine J., Larsen, Paul D.
Publicado 2018

Mouse Survival Motor Neuron Alleles That Mimic SMN2 Splicing and Are Inducible Rescue Embryonic Lethality Early in Development but Not Late

por Hammond, Suzan M., Gogliotti, Rocky G., Rao, Vamshi, Beauvais, Ariane, Kothary, Rashmi, DiDonato, Christine J.
Publicado 2010

Axonal polyneuropathy and ataxia in children: consider Perrault Syndrome, a case report

por Munson, Hannah E., De Simone, Lenika, Schwaede, Abigail, Bhatia, Avanti, Mithal, Divakar S., Young, Nancy, Kuntz, Nancy, Rao, Vamshi K.
Publicado 2023

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

por 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.
Publicado 2023

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

por 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.
Publicado 2022

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy

por 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.
Publicado 2022

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

por 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.
Publicado 2020

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

por 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
Publicado 2016