Quality of life and subjective symptom impact in Japanese patients with myotonic dystrophy type 1 by Haruo Fujino, Toshio Saito, Masanori P. Takahashi, Hiroto Takada, Takahiro Nakayama, Osamu Imura, Tsuyoshi Matsumura

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Mature Myotubes Generated From Human-Induced Pluripotent Stem Cells Without Forced Gene Expression by Kei Fujiwara, Risa Yamamoto, Tomoya Kubota, Atsutoshi Tazumi, Tomoka Sabuta, Masanori P. Takahashi, Hidetoshi Sakurai

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Optical measurement of gating pore currents in hypokalemic periodic paralysis model cells by Tomoya Kubota, Satoe Takahashi, Risa Yamamoto, Ruka Sato, Aya Miyanooto, Reina Yamamoto, Kosuke Yamauchi, Kazuaki Homma, Masanori P. Takahashi

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Aberrant Myokine Signaling in Congenital Myotonic Dystrophy by Masayuki Nakamori, Kohei Hamanaka, James D. Thomas, Eric T. Wang, Yukiko K. Hayashi, Masanori P. Takahashi, Maurice S. Swanson, Ichizo Nishino, Hideki Mochizuki

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Muscleblind-like 1 knockout mice reveal novel splicing defects in the myotonic dystrophy brain. by Koichi Suenaga, Kuang-Yung Lee, Masayuki Nakamori, Yoshiki Tatsumi, Masanori P Takahashi, Harutoshi Fujimura, Kenji Jinnai, Hiroo Yoshikawa, Hongqing Du, Manuel Ares, Maurice S Swanson, Takashi Kimura

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Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trialResearch in context by Masayuki Nakamori, Daisaku Nakatani, Tomoharu Sato, Yuhei Hasuike, Seiko Kon, Toshio Saito, Harumasa Nakamura, Masanori P. Takahashi, Eisuke Hida, Hirofumi Komaki, Tsuyoshi Matsumura, Hiroto Takada, Hideki Mochizuki

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Straightjacket/α2δ3 deregulation is associated with cardiac conduction defects in myotonic dystrophy type 1 by Emilie Auxerre-Plantié, Masayuki Nakamori, Yoan Renaud, Aline Huguet, Caroline Choquet, Cristiana Dondi, Lucile Miquerol, Masanori P Takahashi, Geneviève Gourdon, Guillaume Junion, Teresa Jagla, Monika Zmojdzian, Krzysztof Jagla

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Cardiac Conduction Disorders as Markers of Cardiac Events in Myotonic Dystrophy Type 1 by Hideki Itoh, Takashi Hisamatsu, Takuhisa Tamura, Kazuhiko Segawa, Toshiaki Takahashi, Hiroto Takada, Satoshi Kuru, Chizu Wada, Mikiya Suzuki, Shugo Suwazono, Shingo Sasaki, Ken Okumura, Minoru Horie, Masanori P. Takahashi, Tsuyoshi Matumura

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The expanding phenotype of hypokalemic periodic paralysis in a Japanese family with p.Val876Glu mutation in CACNA1S by Mari Kurokawa, Michiko Torio, Kazuhiro Ohkubo, Vlad Tocan, Noriko Ohyama, Naoko Toda, Kanako Ishii, Kei Nishiyama, Yuichi Mushimoto, Ryuichi Sakamoto, Maki Nakaza, Riho Horie, Tomoya Kubota, Masanori P. Takahashi, Yasunari Sakai, Masatoshi Nomura, Shouichi Ohga

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Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan by Madoka Mori-Yoshimura, Naoki Suzuki, Masahisa Katsuno, Masanori P. Takahashi, Satoshi Yamashita, Yasushi Oya, Atsushi Hashizume, Shinichiro Yamada, Masayuki Nakamori, Rumiko Izumi, Masaaki Kato, Hitoshi Warita, Maki Tateyama, Hiroshi Kuroda, Ryuta Asada, Takuhiro Yamaguchi, Ichizo Nishino, Masashi Aoki

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The practice of active patient involvement in rare disease research using ICT: experiences and lessons from the RUDY JAPAN project by Nao Hamakawa, Atsushi Kogetsu, Moeko Isono, Chisato Yamasaki, Shirou Manabe, Toshihiro Takeda, Kazumasa Iwamoto, Tomoya Kubota, Joe Barrett, Nathanael Gray, Alison Turner, Harriet Teare, Yukie Imamura, Beverley Anne Yamamoto, Jane Kaye, Michihiro Hide, Masanori P. Takahashi, Yasushi Matsumura, Muhammad Kassim Javaid, Kazuto Kato

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rbFOX1/MBNL1 competition for CCUG RNA repeats binding contributes to myotonic dystrophy type 1/type 2 differences by Chantal Sellier, Estefanía Cerro-Herreros, Markus Blatter, Fernande Freyermuth, Angeline Gaucherot, Frank Ruffenach, Partha Sarkar, Jack Puymirat, Bjarne Udd, John W. Day, Giovanni Meola, Guillaume Bassez, Harutoshi Fujimura, Masanori P. Takahashi, Benedikt Schoser, Denis Furling, Ruben Artero, Frédéric H. T. Allain, Beatriz Llamusi, Nicolas Charlet-Berguerand

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Enhancing evidence-informed policymaking in medicine and healthcare: stakeholder involvement in the Commons Project for rare diseases in Japan by Atsushi Kogetsu, Moeko Isono, Tatsuki Aikyo, Junichi Furuta, Dai Goto, Nao Hamakawa, Michihiro Hide, Risa Hori, Noriko Ikeda, Keiko Inoi, Naomi Kawagoe, Tomoya Kubota, Shirou Manabe, Yasushi Matsumura, Koji Matsuyama, Tomoko Nakai, Ikuko Nakao, Yuki Saito, Midori Senoo, Masanori P. Takahashi, Toshihiro Takeda, Megumi Takei, Katsuto Tamai, Akio Tanaka, Yasuhiro Torashima, Yuya Tsuchida, Chisato Yamasaki, Beverley Anne Yamamoto, Kazuto Kato

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Correction to: Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease by Libby Wood, Guillaume Bassez, Corinne Bleyenheuft, Craig Campbell, Louise Cossette, Aura Cecilia Jimenez-Moreno, Yi Dai, Hugh Dawkins, Jordi Díaz-Manera, Celine Dogan, Rasha el Sherif, Barbara Fossati, Caroline Graham, James Hilbert, Kristinia Kastreva, En Kimura, Lawrence Korngut, Anna Kostera-Pruszczyk, Christopher Lindberg, Bjorn Lindvall, Elizabeth Luebbe, Anna Lusakowska, Radim Mazanec, Giovani Meola, Liannna Orlando, Masanori P. Takahashi, Stojan Peric, Jack Puymirat, Vidosava Rakocevic-Stojanovic, Miriam Rodrigues, Richard Roxburgh, Benedikt Schoser, Sonia Segovia, Andriy Shatillo, Simone Thiele, Ivailo Tournev, Baziel van Engelen, Stanislav Vohanka, Hanns Lochmüller

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