Literature DB >> 33259859

Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.

Deepa H Chand1, Craig Zaidman2, Kapil Arya3, Rachel Millner3, Michelle A Farrar4, Fiona E Mackie4, Natalie L Goedeker2, Vikas R Dharnidharka2, Raja Dandamudi2, Sandra P Reyna5.   

Abstract

Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy following onasemnogene abeparvovec. Early recognition and treatment of drug-induced thrombotic microangiopathy may lessen mortality and morbidity.
Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.

Entities:  

Keywords:  gene therapy; hemolytic uremic syndrome; safety; survival motor neuron

Year:  2020        PMID： 33259859     DOI： 10.1016/j.jpeds.2020.11.054

Source DB:  PubMed          Journal:  J Pediatr        ISSN： 0022-3476            Impact factor:   4.406


  19 in total

Review 1.  Spinal muscular atrophy.

Authors:  Eugenio Mercuri; Charlotte J Sumner; Francesco Muntoni; Basil T Darras; Richard S Finkel
Journal:  Nat Rev Dis Primers       Date:  2022-08-04       Impact factor: 65.038

Review 2.  Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies.

Authors:  Claudia A Chiriboga
Journal:  Paediatr Drugs       Date:  2022-08-27       Impact factor: 3.930

Review 3.  Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich's Ataxia.

Authors:  Anusha Sivakumar; Stephanie Cherqui
Journal:  Front Genome Ed       Date:  2022-05-17

4.  Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.

Authors:  Kevin A Strauss; Michelle A Farrar; Francesco Muntoni; Kayoko Saito; Jerry R Mendell; Laurent Servais; Hugh J McMillan; Richard S Finkel; Kathryn J Swoboda; Jennifer M Kwon; Craig M Zaidman; Claudia A Chiriboga; Susan T Iannaccone; Jena M Krueger; Julie A Parsons; Perry B Shieh; Sarah Kavanagh; Melissa Wigderson; Sitra Tauscher-Wisniewski; Bryan E McGill; Thomas A Macek
Journal:  Nat Med       Date:  2022-06-17       Impact factor: 87.241

Review 5.  Durability of transgene expression after rAAV gene therapy.

Authors:  Manish Muhuri; Daniel I Levy; Martin Schulz; Douglas McCarty; Guangping Gao
Journal:  Mol Ther       Date:  2022-03-10       Impact factor: 12.910

Review 6.  Considerations for Systemic Use of Gene Therapy.

Authors:  Barry J Byrne; Manuela Corti; Francesco Muntoni
Journal:  Mol Ther       Date:  2021-01-14       Impact factor: 11.454

7.  Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.

Authors:  Jerry R Mendell; Samiah A Al-Zaidy; Kelly J Lehman; Markus McColly; Linda P Lowes; Lindsay N Alfano; Natalie F Reash; Megan A Iammarino; Kathleen R Church; Aaron Kleyn; Matthew N Meriggioli; Richard Shell
Journal:  JAMA Neurol       Date:  2021-07-01       Impact factor: 18.302

8.  Assuring long-term safety of highly effective gene-modulating therapeutics for rare diseases.

Authors:  Thomas O Crawford; Charlotte J Sumner
Journal:  J Clin Invest       Date:  2021-08-02       Impact factor: 19.456

9.  Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy.

Authors:  John W Day; Jerry R Mendell; Eugenio Mercuri; Richard S Finkel; Kevin A Strauss; Aaron Kleyn; Sitra Tauscher-Wisniewski; Francis Fonyuy Tukov; Sandra P Reyna; Deepa H Chand
Journal:  Drug Saf       Date:  2021-08-12       Impact factor: 5.606

10.  Gene therapy for spinal muscular atrophy: the Qatari experience.

Authors:  Hossamaldein Gaber Ali; Khalid Ibrahim; Mahmoud Fawzi Elsaid; Reem Babiker Mohamed; Mahmoud I A Abeidah; Azhar Othman Al Rawwas; Khaled Elshafey; Hajer Almulla; Karen El-Akouri; Mariam Almulla; Amna Othman; Sara Musa; Fatma Al-Mesaifri; Rehab Ali; Noora Shahbeck; Mariam Al-Mureikhi; Reem Alsulaiman; Saad Alkaabi; Tawfeg Ben-Omran
Journal:  Gene Ther       Date:  2021-07-19       Impact factor: 5.250
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