Literature DB >> 34895502

Advanced therapeutic strategy for hereditary neuromuscular diseases.

Wuh-Liang Hwu1, Shin-Ichi Muramatsu2, Yin-Hsiu Chien3, Barry J Byrne4.   

Abstract

Entities:  

Mesh：

Year:  2021        PMID： 34895502      PMCID： PMC8753517          DOI： 10.1016/j.ymthe.2021.12.004

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  9 in total

1.  Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy.

Authors:  James M Wilson; Terence R Flotte
Journal:  Hum Gene Ther       Date:  2020-07-01       Impact factor: 5.695

2.  Spinal muscular atrophy: a timely review.

Authors:  Stephen J Kolb; John T Kissel
Journal:  Arch Neurol       Date:  2011-04-11

3.  Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.

Authors:  Imke Anne Maartje Ditters; Hidde Harmen Huidekoper; Michelle Elisabeth Kruijshaar; Dimitris Rizopoulos; Andreas Hahn; Tiziana Enrica Mongini; François Labarthe; Marine Tardieu; Brigitte Chabrol; Anais Brassier; Rossella Parini; Giancarlo Parenti; Nadine Anna Maria Elisabeth van der Beek; Ans Tjitske van der Ploeg; Johanna Maria Pieternel van den Hout
Journal:  Lancet Child Adolesc Health       Date:  2021-11-22

4.  Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.

Authors:  Claudia Weiß; Andreas Ziegler; Lena-Luise Becker; Jessika Johannsen; Heiko Brennenstuhl; Gudrun Schreiber; Marina Flotats-Bastardas; Corinna Stoltenburg; Hans Hartmann; Sabine Illsinger; Jonas Denecke; Astrid Pechmann; Wolfgang Müller-Felber; Katharina Vill; Astrid Blaschek; Martin Smitka; Lieske van der Stam; Katja Weiss; Benedikt Winter; Klaus Goldhahn; Barbara Plecko; Veronka Horber; Günther Bernert; Ralf A Husain; Christian Rauscher; Regina Trollmann; Sven F Garbade; Andreas Hahn; Maja von der Hagen; Angela M Kaindl
Journal:  Lancet Child Adolesc Health       Date:  2021-10-29

5.  Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.

Authors:  Eugenio Mercuri; Francesco Muntoni; Giovanni Baranello; Riccardo Masson; Odile Boespflug-Tanguy; Claudio Bruno; Stefania Corti; Aurore Daron; Nicolas Deconinck; Laurent Servais; Volker Straub; Haojun Ouyang; Deepa Chand; Sitra Tauscher-Wisniewski; Nuno Mendonca; Arseniy Lavrov
Journal:  Lancet Neurol       Date:  2021-10       Impact factor: 44.182

6.  Pompe disease in infants: improving the prognosis by newborn screening and early treatment.

Authors:  Yin-Hsiu Chien; Ni-Chung Lee; Beth L Thurberg; Shu-Chuan Chiang; Xiaokui Kate Zhang; Joan Keutzer; Ai-Chu Huang; Mei-Hwan Wu; Pei-Hsin Huang; Fuu-Jen Tsai; Yuan-Tsong Chen; Wuh-Liang Hwu
Journal:  Pediatrics       Date:  2009-12       Impact factor: 7.124

7.  A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.

Authors:  Torayuki Okuyama; Yoshikatsu Eto; Norio Sakai; Kimitoshi Nakamura; Tatsuyoshi Yamamoto; Mariko Yamaoka; Toshiaki Ikeda; Sairei So; Kazunori Tanizawa; Hiroyuki Sonoda; Yuji Sato
Journal:  Mol Ther       Date:  2020-09-30       Impact factor: 11.454

8.  A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.

Authors:  Giang N Nguyen; John K Everett; Samita Kafle; Aoife M Roche; Hayley E Raymond; Jacob Leiby; Christian Wood; Charles-Antoine Assenmacher; Elizabeth P Merricks; C Tyler Long; Haig H Kazazian; Timothy C Nichols; Frederic D Bushman; Denise E Sabatino
Journal:  Nat Biotechnol       Date:  2020-11-16       Impact factor: 54.908

9.  Gene therapy in the putamen for curing AADC deficiency and Parkinson's disease.

Authors:  Paul Wuh-Liang Hwu; Karl Kiening; Irina Anselm; David R Compton; Takeshi Nakajima; Thomas Opladen; Phillip L Pearl; Agathe Roubertie; Thomas Roujeau; Shin-Ichi Muramatsu
Journal:  EMBO Mol Med       Date:  2021-08-23       Impact factor: 12.137
  9 in total

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