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Newborn Screening for Spinal Muscular Atrophy in Osaka -Challenges in a Japanese Pilot Study

Tomokazu Kimizu,Shinobu Ida,Keisuke Oki, Morimasa Shima,Shizuka Nishimoto,Ken Nakajima,Tae Ikeda,Yukiko Mogami,Keiko Yanagihara,Keiko Matsuda,Eriko Nishi,Yuiko Hasegawa,Masatoshi Nozaki, Hiroshi Fujita,Akemi Irie, Toru Katayama,Nobuhiko Okamoto,Kohsuke Imai,Hisahide Nishio,Yasuhiro Suzuki

BRAIN & DEVELOPMENT（2023）

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Key words
Newborn screening,Spinal muscular atrophy,Early treatment,Survival motor neuron gene,Gene replacement therapy
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