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Combination molecular therapies for type 1 spinal muscular atrophy
Author(s) -
Harada Yohei,
Rao Vamshi K.,
Arya Kapil,
Kuntz Nancy L.,
DiDonato Christine J.,
NapchanPomerantz Galia,
Agarwal Amit,
Stefans Vikki,
Katsuno Masahisa,
Veerapandiyan Aravindhan
Publication year - 2020
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.27034
Subject(s) - medicine , spinal muscular atrophy , corticosteroid , atrophy , combination therapy , adverse effect , muscle biopsy , biopsy , liver biopsy , surgery , disease
Abstract Background 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. Conclusions 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.