Результати пошуку - Amy D. Harper
- Показ 1 - 5 результатів із 5
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1
Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy за авторством Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly, Amy D. Harper, Jean K. Mah, Craig M. McDonald, Edward C. Smith, Craig M. Zaidman, Tomoyuki Nakagawa, Eric P. Hoffman
Опубліковано 2022Artigo -
2
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study за авторством Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly, Amy D. Harper, Jean K. Mah, Craig M. McDonald, Edward C. Smith, Craig M. Zaidman, Tomoyuki Nakagawa, Eric P. Hoffman
Опубліковано 2023Artigo -
3
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping за авторством 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
Опубліковано 2020Artigo -
4
Spinocerebellar ataxia type 29 due to mutations in ITPR1: a case series and review of this emerging congenital ataxia за авторством Jessica L. Zambonin, Allison Bellomo, Hilla Ben‐Pazi, David B. Everman, Lee M. Frazer, Michael T. Geraghty, Amy D. Harper, Julie R. Jones, Benjamin Kamien, Kristin D. Kernohan, Mary Kay Koenig, Matthew A. Lines, Elizabeth E. Palmer, Randal Richardson, Reeval Segel, Mark A. Tarnopolsky, Jason Vanstone, Melissa Gibbons, Abigail Collins, Brent L. Fogel, Tracy Dudding‐Byth, Kym M. Boycott
Опубліковано 2017Revisão -
5
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy за авторством Michela Guglieri, Paula R. Clemens, Seth J. Perlman, Edward C. Smith, Iain Horrocks, Richard S. Finkel, Jean K. Mah, Nicolas Deconinck, Nathalie Goemans, Jana Haberlová, Volker Straub, L. Mengle-Gaw, Benjamin D. Schwartz, Amy D. Harper, Perry B. Shieh, Liesbeth De Waele, Diana Castro, Michelle Yang, Monique M. Ryan, Craig M. McDonald, M. Tulinius, Richard Webster, Hugh J. McMillan, Nancy L. Kuntz, Vashmi K. Rao, Giovanni Baranello, Stefan Spinty, Anne‐Marie Childs, Annie M. Sbrocchi, Kathryn Selby, Migvis Monduy, Yoram Nevo, Juan J. Vílchez, Andres Nascimento-Osorio, Erik H. Niks, Imelda J. M. de Groot, Marina Katsalouli, M. James, John van den Anker, Jesse M. Damsker, Alexandra Ahmet, Leanne M. Ward, Mark Jaros, Phil Shale, Utkarsh J. Dang, Eric P. Hoffman
Опубліковано 2022Artigo
Інструменти для пошуку:
Пов'язані теми
Medicine
Pediatrics
Duchenne muscular dystrophy
Internal medicine
Adverse effect
Biology
Clinical trial
Gene
Muscular dystrophy
Pathology
Physical therapy
Age of onset
Alternative medicine
Alternative splicing
Ataxia
Atrophy
Biochemistry
Cerebellar ataxia
Clinical endpoint
Cohort
Computer science
Disease
Dystrophin
Exon
Exon skipping
Extension (predicate logic)
Genetics
Human genetics
Hypotonia
Missense mutation