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Items: 1 to 20 of 67

1.

Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease.

Rao VK, Kapp D, Schroth M.

J Manag Care Spec Pharm. 2018 Dec;24(12-a Suppl):S3-S16. doi: 10.18553/jmcp.2018.24.12-a.s3. Review.

2.

Spinal muscular atrophy 5Q - Treatment with nusinersen.

Brazilian Medical Association, Silvinato A, Bernardo WM.

Rev Assoc Med Bras (1992). 2018 Jun;64(6):484-491. doi: 10.1590/1806-9282.64.06.484. Review.

3.

Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps.

Gidaro T, Servais L.

Dev Med Child Neurol. 2019 Jan;61(1):19-24. doi: 10.1111/dmcn.14027. Epub 2018 Sep 17. Review.

PMID:
30221755
4.

Prenatal aspects in spinal muscular atrophy: From early detection to early presymptomatic intervention.

Tizzano EF, Zafeiriou D.

Eur J Paediatr Neurol. 2018 Nov;22(6):944-950. doi: 10.1016/j.ejpn.2018.08.009. Epub 2018 Sep 3. Review.

PMID:
30219357
5.

Measuring quality of life in children with spinal muscular atrophy: a systematic literature review.

Vaidya S, Boes S.

Qual Life Res. 2018 Dec;27(12):3087-3094. doi: 10.1007/s11136-018-1945-x. Epub 2018 Jul 24. Review. Erratum in: Qual Life Res. 2018 Sep 14;:.

PMID:
30043243
6.

RNP Assembly Defects in Spinal Muscular Atrophy.

Price PL, Morderer D, Rossoll W.

Adv Neurobiol. 2018;20:143-171. doi: 10.1007/978-3-319-89689-2_6. Review.

PMID:
29916019
7.

Mechanism of Splicing Regulation of Spinal Muscular Atrophy Genes.

Singh RN, Singh NN.

Adv Neurobiol. 2018;20:31-61. doi: 10.1007/978-3-319-89689-2_2. Review.

8.

Therapeutic advances in 5q-linked spinal muscular atrophy.

Reed UC, Zanoteli E.

Arq Neuropsiquiatr. 2018 Apr;76(4):265-272. doi: 10.1590/0004-282x20180011. Review.

9.

Spinal muscular atrophy.

Arnold ES, Fischbeck KH.

Handb Clin Neurol. 2018;148:591-601. doi: 10.1016/B978-0-444-64076-5.00038-7. Review.

PMID:
29478602
10.

Pathogenic commonalities between spinal muscular atrophy and amyotrophic lateral sclerosis: Converging roads to therapeutic development.

Bowerman M, Murray LM, Scamps F, Schneider BL, Kothary R, Raoul C.

Eur J Med Genet. 2018 Nov;61(11):685-698. doi: 10.1016/j.ejmg.2017.12.001. Epub 2017 Dec 5. Review.

PMID:
29313812
11.

Treatment Advances in Spinal Muscular Atrophy.

Bharucha-Goebel D, Kaufmann P.

Curr Neurol Neurosci Rep. 2017 Oct 6;17(11):91. doi: 10.1007/s11910-017-0798-y. Review.

12.

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape.

Wood MJA, Talbot K, Bowerman M.

Hum Mol Genet. 2017 Oct 1;26(R2):R151-R159. doi: 10.1093/hmg/ddx215. Review.

PMID:
28977438
13.

Nusinersen for the treatment of spinal muscular atrophy.

Chiriboga CA.

Expert Rev Neurother. 2017 Oct;17(10):955-962. doi: 10.1080/14737175.2017.1364159. Epub 2017 Sep 8. Review.

PMID:
28884620
14.

Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy.

Paton DM.

Drugs Today (Barc). 2017 Jun;53(6):327-337. doi: 10.1358/dot.2017.53.6.2652413. Review.

PMID:
28799578
15.

Therapeutic strategies for spinal muscular atrophy: SMN and beyond.

Bowerman M, Becker CG, Yáñez-Muñoz RJ, Ning K, Wood MJA, Gillingwater TH, Talbot K; UK SMA Research Consortium.

Dis Model Mech. 2017 Aug 1;10(8):943-954. doi: 10.1242/dmm.030148. Review.

16.

Spinal muscular atrophy: A changing phenotype beyond the clinical trials.

Tizzano EF, Finkel RS.

Neuromuscul Disord. 2017 Oct;27(10):883-889. doi: 10.1016/j.nmd.2017.05.011. Epub 2017 May 17. Review.

PMID:
28757001
17.

Nusinersen: The First Option Beyond Supportive Care for Spinal Muscular Atrophy.

Maharshi V, Hasan S.

Clin Drug Investig. 2017 Sep;37(9):807-817. doi: 10.1007/s40261-017-0557-5. Review.

PMID:
28755059
18.

Management of neuromuscular diseases and spinal muscular atrophy in Latin America.

Monges S, Rosa AL.

Gene Ther. 2017 Sep;24(9):578-580. doi: 10.1038/gt.2017.68. Epub 2017 Jul 28. Review.

PMID:
28753203
19.

Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review.

Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, Cook SF, Lochmüller H.

Orphanet J Rare Dis. 2017 Jul 4;12(1):124. doi: 10.1186/s13023-017-0671-8. Review.

20.

Advances in understanding the role of disease-associated proteins in spinal muscular atrophy.

Hosseinibarkooie S, Schneider S, Wirth B.

Expert Rev Proteomics. 2017 Jul;14(7):581-592. doi: 10.1080/14789450.2017.1345631. Epub 2017 Jun 30. Review.

PMID:
28635376

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