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Items: 1 to 20 of 67

1.

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.

PMID:
28983837
2.

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
3.

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
4.

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.

5.

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
6.

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
7.

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.

8.

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
9.

Inherited Paediatric Motor Neuron Disorders: Beyond Spinal Muscular Atrophy.

Teoh HL, Carey K, Sampaio H, Mowat D, Roscioli T, Farrar M.

Neural Plast. 2017;2017:6509493. doi: 10.1155/2017/6509493. Epub 2017 May 28. Review.

10.

Diagnosis and New Treatment Avenues in Spinal Muscular Atrophy.

Pechmann A, Kirschner J.

Neuropediatrics. 2017 Aug;48(4):273-281. doi: 10.1055/s-0037-1603517. Epub 2017 Jun 1. Review. No abstract available.

PMID:
28571100
11.

Therapeutic approaches for spinal muscular atrophy (SMA).

Scoto M, Finkel RS, Mercuri E, Muntoni F.

Gene Ther. 2017 Sep;24(9):514-519. doi: 10.1038/gt.2017.45. Epub 2017 May 31. Review.

PMID:
28561813
12.

Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy.

Jablonka S, Sendtner M.

Gene Ther. 2017 Sep;24(9):506-513. doi: 10.1038/gt.2017.46. Epub 2017 May 30. Review.

PMID:
28556834
13.

How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy.

Singh NN, Howell MD, Androphy EJ, Singh RN.

Gene Ther. 2017 Sep;24(9):520-526. doi: 10.1038/gt.2017.34. Epub 2017 May 9. Review.

14.

Adult-onset spinal muscular atrophy: An update.

Juntas Morales R, Pageot N, Taieb G, Camu W.

Rev Neurol (Paris). 2017 May;173(5):308-319. doi: 10.1016/j.neurol.2017.03.015. Epub 2017 Apr 26. Review.

PMID:
28456383
15.

Cardiac pathology in spinal muscular atrophy: a systematic review.

Wijngaarde CA, Blank AC, Stam M, Wadman RI, van den Berg LH, van der Pol WL.

Orphanet J Rare Dis. 2017 Apr 11;12(1):67. doi: 10.1186/s13023-017-0613-5. Review.

16.

Spinal muscular atrophy: Factors that modulate motor neurone vulnerability.

Tu WY, Simpson JE, Highley JR, Heath PR.

Neurobiol Dis. 2017 Jun;102:11-20. doi: 10.1016/j.nbd.2017.01.011. Epub 2017 Feb 2. Review.

PMID:
28161391
17.

Linking amyotrophic lateral sclerosis and spinal muscular atrophy through RNA-transcriptome homeostasis: a genomics perspective.

Gama-Carvalho M, L Garcia-Vaquero M, R Pinto F, Besse F, Weis J, Voigt A, Schulz JB, De Las Rivas J.

J Neurochem. 2017 Apr;141(1):12-30. doi: 10.1111/jnc.13945. Epub 2017 Feb 28. Review.

18.

Emerging therapies and challenges in spinal muscular atrophy.

Farrar MA, Park SB, Vucic S, Carey KA, Turner BJ, Gillingwater TH, Swoboda KJ, Kiernan MC.

Ann Neurol. 2017 Mar;81(3):355-368. doi: 10.1002/ana.24864. Epub 2017 Feb 17. Review.

19.

Spinal Muscular Atrophy: More than a Disease of Motor Neurons?

Nash LA, Burns JK, Chardon JW, Kothary R, Parks RJ.

Curr Mol Med. 2016;16(9):779-792. doi: 10.2174/1566524016666161128113338. Review.

PMID:
27894243
20.

Spinal muscular atrophy associated with progressive myoclonus epilepsy.

Topaloglu H, Melki J.

Epileptic Disord. 2016 Sep 1;18(S2):128-134. Review.

PMID:
27647482

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