Search Results - Francesco Muntoni

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  1. 1
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    The authors reply: Letter on: "Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy" by Zhou et al. by Haiyan Zhou, Francesco Muntoni

    Published 2020
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  2. 2
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    Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy by Elena Marrosu, Pierpaolo Ala, Francesco Muntoni, Haiyan Zhou

    Published 2017
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  3. 3
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    Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy by Francesco Catapano, Irina Zaharieva, Mariacristina Scoto, Elena Marrosu, Jennifer Morgan, Francesco Muntoni, Haiyan Zhou

    Published 2016
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  4. 4
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    Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD by Sara Aguti, Véronique Bolduc, Pierpaolo Ala, Mark Turmaine, Carsten G. Bönnemann, Francesco Muntoni, Haiyan Zhou

    Published 2020
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  5. 5
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    Screening of Neonatal UK Dried Blood Spots Using a Duplex SMN1 Screening Assay by Stuart P. Adams, Emma Gravett, Natalie Kent, Susanne Kricke, Adeboye Ifederu, Mariacristina Scoto, Salma Samsuddin, Francesco Muntoni

    Published 2021
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  6. 6
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    Targeted Exon Skipping to Address "Leaky" Mutations in the Dystrophin Gene by Sue Fletcher, Carl F. Adkin, Penny Meloni, Brenda Wong, Francesco Muntoni, Ryszard Kole, Clayton Fragall, Kane Greer, Russell Johnsen, Steve D. Wilton

    Published 2012
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  7. 7
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    Comparative proteomic analyses of Duchenne muscular dystrophy and Becker muscular dystrophy muscles: changes contributing to preserve muscle function in Becker muscular dystrophy p... by Daniele Capitanio, Manuela Moriggi, Enrica Torretta, Pietro Barbacini, Sara De Palma, Agnese Viganò, Hanns Lochmüller, Francesco Muntoni, Alessandra Ferlini, Marina Mora, Cecilia Gelfi

    Published 2020
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  8. 8
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    Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice by Amel Saoudi, Sacha Barberat, Olivier le Coz, Ophélie Vacca, Mathilde Doisy Caquant, Thomas Tensorer, Eric Sliwinski, Luis Garcia, Francesco Muntoni, Cyrille Vaillend, Aurélie Goyenvalle

    Published 2023
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  9. 9
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    Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy by Mirko Signorelli, Burcu Ayoglu, Camilla Johansson, Hanns Lochmüller, Volker Straub, Francesco Muntoni, Erik Niks, Roula Tsonaka, Anja Persson, Annemieke Aartsma‐Rus, Peter Nilsson, Cristina Al‐Khalili Szigyarto, Pietro Spitali

    Published 2020
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  10. 10
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    Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy by Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni

    Published 2020
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  11. 11
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    Cardiac Manifestations of Myotonic Dystrophy in a Pediatric Cohort by Laia Brunet Garcia, Laia Brunet Garcia, Ankita Hajra, Ella Field, Joseph Wacher, Helen Walsh, Gabrielle Norrish, Adnan Manzur, Francesco Muntoni, Pinki Munot, Stephanie Robb, Rosaline Quinlivan, Mariacristina Scoto, Giovanni Baranello, Anna Sarkozy, Luke Starling, Juan Pablo Kaski, Elena Cervi

    Published 2022
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  12. 12
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    Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy by Mary Chesshyre, Deborah Ridout, Yasumasa Hashimoto, Yoko Ookubo, Silvia Torelli, Kate Maresh, Valeria Ricotti, Lianne Abbott, Vandana Ayyar Gupta, Marion Main, Giulia Ferrari, Anna Kowala, Yung‐Yao Lin, Francesco Saverio Tedesco, Mariacristina Scoto, Giovanni Baranello, Adnan Manzur, Yoshitsugu Aoki, Francesco Muntoni

    Published 2022
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  13. 13
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    Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies by Pietro Spitali, Kristina Hettne, Roula Tsonaka, Mohammed Charrout, Janneke van denBergen, Zaïda Koeks, Hermien E. Kan, Melissa T. Hooijmans, Andreas Roos, Volker Straub, Francesco Muntoni, Cristina Al‐Khalili‐Szigyarto, Marleen J.A. Koel‐Simmelink, Charlotte E. Teunissen, Hanns Lochmüller, Erik H. Niks, Annemieke Aartsma‐Rus

    Published 2018
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