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Therapy for Spinal Muscular Atrophy.

作者信息

Mendell Jerry R

机构信息

Nationwide Children’s Hospital, Columbus, OH

出版信息

N Engl J Med. 2018 Feb 1;378(5):487. doi: 10.1056/NEJMc1715769.

DOI:10.1056/NEJMc1715769

PMID:29394473

Abstract

摘要

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相似文献

Therapy for Spinal Muscular Atrophy.

N Engl J Med. 2018 Feb 1;378(5):487. doi: 10.1056/NEJMc1715769.

[On the problem of spinal muscular atrophy resembling progressive muscular dystrophy (Kugelberg-Welander's juvenile muscular atrophy)].

Neurol Neurochir Psychiatr Pol. 1962 Sep-Oct;12:669-73.

Therapy for Spinal Muscular Atrophy.

N Engl J Med. 2018 Feb 1;378(5):487-488. doi: 10.1056/NEJMc1715769.

Therapy for Spinal Muscular Atrophy.

N Engl J Med. 2018 Feb 1;378(5):487. doi: 10.1056/NEJMc1715769.

Familial progressive bulbar-spinal muscular atrophy.

Neurology. 1960 Mar;10:295-305. doi: 10.1212/wnl.10.3.295.

Stakeholder collaboration for spinal muscular atrophy therapy development.

Lancet Neurol. 2017 Apr;16(4):264. doi: 10.1016/S1474-4422(17)30041-8.

Another milestone in childhood spinal muscular atrophy.

Lancet Neurol. 2017 Jul;16(7):491-492. doi: 10.1016/S1474-4422(17)30121-7. Epub 2017 Apr 28.

Genetic therapies for spinal muscular atrophy type 1.

Lancet Neurol. 2018 Feb;17(2):111-112. doi: 10.1016/S1474-4422(17)30436-2. Epub 2017 Dec 8.

Distal infantile spinal muscular atrophy associated with paralysis of the diaphragm: a variant of infantile spinal muscular atrophy.

Am J Med Genet. 1989 Jul;33(3):328-35. doi: 10.1002/ajmg.1320330309.

Infantile spinal progressive muscular atrophy in twins.

Can Med Assoc J. 1951 Oct;65(4):342-3.

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Mol Ther Oncolytics. 2019 Oct 7;15:166-177. doi: 10.1016/j.omto.2019.10.001. eCollection 2019 Dec 20.

Regenerative medicine: the red planet for clinicians.

Intern Emerg Med. 2019 Sep;14(6):911-921. doi: 10.1007/s11739-019-02126-z. Epub 2019 Jun 15.