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

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

Longitudinal Assessment of Timed Function Tests Over Time in Ambulatory Individuals with SMA Treated with Nusinersen.

Krosschell, Kristin J; Brown, Laurey; Hoffman, Katie; Zumpf, Katelyn B; Munson, Hannah; Bidwell, Jessa; Schulte, Duncan P; Schwaede, Abigail N; Buehner, Amber N; DiDonato, Christine J; Kuntz, Nancy L; Rao, Vamshi K

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

Palliative care in Duchenne muscular dystrophy: Goals of care discussions and beyond.

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

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

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

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

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

Spinal Muscular Atrophy Diagnosed by Newborn Screening.

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

PIGQ glycosylphosphatidylinositol‐anchored protein deficiency: Characterizing the phenotype

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

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

Vamshi K. Rao, Daniel Kapp, Mary Schroth

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