https://onlinelibrary.wiley.com/doi/abs/10.1002/mus.27034
https://doi.org/10.1002/mus.27034
https://pubmed.ncbi.nlm.nih.gov/32710634/
TITLE:
Combination molecular therapies for type 1 spinal muscular atrophy
ALTERNATIVE TITLE:
None
DATE:
Verify – Sat, 25 Jul 2020 06:00:00 -0400
AUTHORS:
Yohei Harada,Vamshi K Rao,Kapil Arya,Nancy L Kuntz,Christine J DiDonato,Galia Napchan-Pomerantz,Amit Agarwal,Vikki Stefans,Masahisa Katsuno,Aravindhan Veerapandiyan
SOURCE:
Muscle & nerve
DESCRIPTION:
INTRODUCTION: Data on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking.
CONTENT:
Muscle Nerve. 2020 Jul 25. doi: 10.1002/mus.27034. Online ahead of print.
ABSTRACT
INTRODUCTION: Data on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking.
METHODS: This was a retrospective study describing our centers’ experiences in treating SMA1 patients with combination therapy.
RESULTS: Five children received nusinersen and onasemnogene abeparvovec-xioi (onasemnogene). Four were receiving nusinersen prior to onasemnogene. Nusinersen was continued in three. Marked liver enzyme elevations resulted in prolonged corticosteroid treatment in two patients with hospitalization and liver biopsy in one; milder liver enzyme elevations were noted in the other two. One patient received onasemnogene first, and then nusinersen. No adverse effects were noted. All patients improved.
DISCUSSION: Combination molecular therapy is tolerated in SMA1 patients. Further studies are needed to determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy. Prolonged corticosteroid use and liver toxicity monitoring may be necessary with onasemnogene therapy. This article is protected by copyright. All rights reserved.
PMID:32710634 | DOI:10.1002/mus.27034
PUBMED ID:
pubmed:32710634
OTHER ID:
pmid:32710634,doi:10.1002/mus.27034
PUBLICATION DATE:
Sat, 25 Jul 2020 06:00:00 -0400
2020-07-26
RETRIEVAL DATE :
07/25/20 06:28PM
LINK – PUBMED:
https://pubmed.ncbi.nlm.nih.gov/32710634/?utm_source=MS-Office&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1l7lJQBQXfTBJkL86rnvYKzafMiKbgcUrlv_X8_D_H5EuRmkjR&fc=20200708141943&ff=20200725182855&v=2.11.3
LINK – DOI:
https://doi.org/10.1002/mus.27034
LINK – FULL TEXT:
Pending
NOTES:
None
