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Genetics in Medicine Open
(Jan 2023)
P260: Survival in eteplirsen-treated vs Duchenne muscular dystrophy natural history patients: An indirect treatment comparison using real-world data
Erin O’Rourke,
Joel Iff,
Nicolae Done,
Ed Tuttle,
Yi Zhong,
Fangzhou Wei,
Basil Darras,
Craig McDonald,
Eugenio Mercuri,
Francesco Muntoni
Affiliations
Erin O’Rourke
Sarepta Therapeutics, Inc., Cambridge, MA
Joel Iff
Sarepta Therapeutics, Inc., Cambridge, MA
Nicolae Done
Analysis Group, Inc., Boston, MA
Ed Tuttle
Analysis Group, Inc., Boston, MA
Yi Zhong
Analysis Group, Inc., Boston, MA
Fangzhou Wei
Analysis Group, Inc., Boston, MA
Basil Darras
Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA
Craig McDonald
University of California Davis Health System, Sacramento, CA
Eugenio Mercuri
Paediatric Neurology and Centro Clinico Nemo, Catholic University, Rome, Italy
Francesco Muntoni
Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, UK
Journal volume & issue
Vol. 1, no. 1
p. 100288
Abstract
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No abstracts available.
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