Literature DB >> 35209987

Recent research on the treatment of spinal muscular atrophy.

Dong-Ling Yang1.   

Abstract

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive muscular weakness and atrophy. SMA, as an inherited disease, is the leading cause of death in infants and young children. Rapid progress has been made in the research field of SMA in recent years, and some related treatment drugs have been successfully approved for marketing. This article reviews the recent research advances in the treatment of SMA.

Entities:  

Keywords:  Gene therapy; Spinal muscular atrophy; Survival motor neuron gene

Mesh：

Year:  2022        PMID： 35209987      PMCID： PMC8884051          DOI： 10.7499/j.issn.1008-8830.2110041

Source DB:  PubMed          Journal:  Zhongguo Dang Dai Er Ke Za Zhi        ISSN： 1008-8830


  36 in total

1.  NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice.

Authors:  Laura Torres-Benito; Svenja Schneider; Roman Rombo; Karen K Ling; Vanessa Grysko; Aaradhita Upadhyay; Natalia L Kononenko; Frank Rigo; C Frank Bennett; Brunhilde Wirth
Journal:  Am J Hum Genet       Date:  2019-06-20       Impact factor: 11.025

2.  The zinc finger protein ZPR1 is a potential modifier of spinal muscular atrophy.

Authors:  Saif Ahmad; Yi Wang; Gouse M Shaik; Arthur H Burghes; Laxman Gangwani
Journal:  Hum Mol Genet       Date:  2012-03-14       Impact factor: 6.150

3.  Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Authors:  Yimin Hua; Kentaro Sahashi; Gene Hung; Frank Rigo; Marco A Passini; C Frank Bennett; Adrian R Krainer
Journal:  Genes Dev       Date:  2010-07-12       Impact factor: 11.361

Review 4.  Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.

Authors:  Alyssa N Calder; Elliot J Androphy; Kevin J Hodgetts
Journal:  J Med Chem       Date:  2016-08-16       Impact factor: 7.446

Review 5.  Olesoxime in neurodegenerative diseases: Scrutinising a promising drug candidate.

Authors:  Jonasz Jeremiasz Weber; Laura Emily Clemensson; Helgi Birgir Schiöth; Huu Phuc Nguyen
Journal:  Biochem Pharmacol       Date:  2019-07-05       Impact factor: 5.858

6.  Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Authors:  Richard S Finkel; Eugenio Mercuri; Basil T Darras; Anne M Connolly; Nancy L Kuntz; Janbernd Kirschner; Claudia A Chiriboga; Kayoko Saito; Laurent Servais; Eduardo Tizzano; Haluk Topaloglu; Már Tulinius; Jacqueline Montes; Allan M Glanzman; Kathie Bishop; Z John Zhong; Sarah Gheuens; C Frank Bennett; Eugene Schneider; Wildon Farwell; Darryl C De Vivo
Journal:  N Engl J Med       Date:  2017-11-02       Impact factor: 91.245

Review 7.  Nusinersen: First Global Approval.

Authors:  Sheridan M Hoy
Journal:  Drugs       Date:  2017-03       Impact factor: 9.546

8.  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

9.  A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy.

Authors:  Doreen Barrett; Sanela Bilic; Yung Chyung; Shaun M Cote; Ryan Iarrobino; Katherine Kacena; Ashish Kalra; Kimberly Long; George Nomikos; Amy Place; James Gordon Still; Leela Vrishabhendra
Journal:  Adv Ther       Date:  2021-05-08       Impact factor: 3.845

10.  Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.

Authors:  Darryl C De Vivo; Enrico Bertini; Kathryn J Swoboda; Wuh-Liang Hwu; Thomas O Crawford; Richard S Finkel; Janbernd Kirschner; Nancy L Kuntz; Julie A Parsons; Monique M Ryan; Russell J Butterfield; Haluk Topaloglu; Tawfeg Ben-Omran; Valeria A Sansone; Yuh-Jyh Jong; Francy Shu; John F Staropoli; Douglas Kerr; Alfred W Sandrock; Christopher Stebbins; Marco Petrillo; Gabriel Braley; Kristina Johnson; Richard Foster; Sarah Gheuens; Ishir Bhan; Sandra P Reyna; Stephanie Fradette; Wildon Farwell
Journal:  Neuromuscul Disord       Date:  2019-09-12       Impact factor: 4.296
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