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Items: 11

1.

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP.

Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332.

2.

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.

Finkel RS, Flanigan KM, Wong B, Bönnemann C, Sampson J, Sweeney HL, Reha A, Northcutt VJ, Elfring G, Barth J, Peltz SW.

PLoS One. 2013 Dec 11;8(12):e81302. doi: 10.1371/journal.pone.0081302. eCollection 2013.

3.

The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence JM, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz S.

Muscle Nerve. 2013 Sep;48(3):343-56. doi: 10.1002/mus.23902. Epub 2013 Jun 26.

4.

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz SW.

Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17.

5.

Corticosteroids in Duchenne muscular dystrophy: major variations in practice.

Griggs RC, Herr BE, Reha A, Elfring G, Atkinson L, Cwik V, McColl E, Tawil R, Pandya S, McDermott MP, Bushby K.

Muscle Nerve. 2013 Jul;48(1):27-31. doi: 10.1002/mus.23831. Epub 2013 Apr 25.

PMID:
23483575
6.

Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences.

Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, Elfring G, Reha A, Barth J, McDonald CM.

Version 2. PLoS Curr. 2012 Jan 25 [revised 2012 Jan 1];4:RRN1297.

7.

Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosis.

Kerem E, Wilschanski M, Miller NL, Pugatsch T, Cohen T, Blau H, Rivlin J, Shoseyov D, Reha A, Constantine S, Ajayi T, Hirawat S, Elfring GL, Peltz SW, Miller LL.

J Cyst Fibros. 2011 May;10(3):193-200. doi: 10.1016/j.jcf.2011.02.003. Epub 2011 Apr 2.

8.

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.

Wilschanski M, Miller LL, Shoseyov D, Blau H, Rivlin J, Aviram M, Cohen M, Armoni S, Yaakov Y, Pugatsch T, Cohen-Cymberknoh M, Miller NL, Reha A, Northcutt VJ, Hirawat S, Donnelly K, Elfring GL, Ajayi T, Kerem E.

Eur Respir J. 2011 Jul;38(1):59-69. doi: 10.1183/09031936.00120910. Epub 2011 Jan 13. Erratum in: Eur Respir J. 2011 Oct;38(4):996. Pugatch, T [corrected to Pugatsch, T].

9.

The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations.

McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Atkinson L, Elfring GL, Reha A, Miller LL.

Muscle Nerve. 2010 Dec;42(6):966-74. doi: 10.1002/mus.21808.

PMID:
21038378
10.

Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.

Sermet-Gaudelus I, Boeck KD, Casimir GJ, Vermeulen F, Leal T, Mogenet A, Roussel D, Fritsch J, Hanssens L, Hirawat S, Miller NL, Constantine S, Reha A, Ajayi T, Elfring GL, Miller LL.

Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. doi: 10.1164/rccm.201001-0137OC. Epub 2010 Jul 9.

PMID:
20622033
11.

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.

McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL.

Muscle Nerve. 2010 Apr;41(4):500-10. doi: 10.1002/mus.21544.

PMID:
19941337

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