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Editorial
. 2021 Mar;63(3):279-281.
doi: 10.1002/mus.27109. Epub 2020 Nov 23.

Combination molecular therapies for spinal muscular atrophy: How much is enough?

Katherine D Mathews  1 , Susan T Iannaccone  2
Affiliations
Editorial

Combination molecular therapies for spinal muscular atrophy: How much is enough?

Katherine D Mathews et al. Muscle Nerve. 2021 Mar.
No abstract available

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References

REFERENCES

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    1. Lee BH, Collins E, Lewis L, et al. Combination therapy with nusinersen and AVXS-101 in SMA type 1. Neurology. 2019;93(14):640-641. https://doi.org/10.1212/WNL.0000000000008207. Epub 2019 Sep 5. PMID: 31488615.
    1. Mercuri E, Lucibello S, Perulli M, et al. Longitudinal natural history of type I spinal muscular atrophy: a critical review. Orphanet J Rare Dis. 2020;15(1):84. https://doi.org/10.1186/s13023-020-01356-1. PMID: 32248834; PMCID: PMC7132885.
    1. Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723-1732. https://doi.org/10.1056/NEJMoa1702752. PMID: 29091570.
    1. Waldrop MA, Karingada C, Storey MA, et al. Gene therapy for spinal muscular atrophy: safety and early outcomes. Pediatrics. 2020;146(3):e20200729. https://doi.org/10.1542/peds.2020-0729. PMID: 32843442.

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