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Items: 1 to 50 of 204

1.

Circulating Plasma microRNAs are Altered with Amyloidosis in a Mouse Model of Alzheimer's Disease.

Ryan MM, Guévremont D, Mockett BG, Abraham WC, Williams JM.

J Alzheimers Dis. 2018;66(2):835-852. doi: 10.3233/JAD-180385.

PMID:
30347618
2.

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR.

Pharmacol Res. 2018 Oct;136:140-150. doi: 10.1016/j.phrs.2018.09.007. Epub 2018 Sep 13.

3.

Falls in paediatric Charcot-Marie-Tooth disease: a 6-month prospective cohort study.

Kennedy RA, Carroll K, Hepworth G, Paterson KL, Ryan MM, McGinley JL.

Arch Dis Child. 2018 Aug 13. pii: archdischild-2018-314890. doi: 10.1136/archdischild-2018-314890. [Epub ahead of print]

PMID:
30104392
4.

What Drives Food Insecurity in Western Australia? How the Perceptions of People at Risk Differ to Those of Stakeholders.

Butcher LM, Ryan MM, O'Sullivan TA, Lo J, Devine A.

Nutrients. 2018 Aug 9;10(8). pii: E1059. doi: 10.3390/nu10081059.

5.

Infantile-Onset Myelin Protein Zero-Related Demyelinating Neuropathy Presenting as an Upper Extremity Monoplegia.

Yiu EM, Wanigasinghe J, Mackay MT, Gonzales M, Nicholson GA, Ryan MM.

Semin Pediatr Neurol. 2018 Jul;26:52-55. doi: 10.1016/j.spen.2017.03.005. Epub 2017 Apr 1.

PMID:
29961519
6.

Genetic, Radiologic, and Clinical Variability in Brown-Vialetto-van Laere Syndrome.

Woodcock IR, Menezes MP, Coleman L, Yaplito-Lee J, Peters H, White SM, Stapleton R, Phelan DG, Chong B, Lunke S, Stark Z, Pitt J, Ryan MM, Robertson C, Yiu EM.

Semin Pediatr Neurol. 2018 Jul;26:2-9. doi: 10.1016/j.spen.2017.03.001. Epub 2017 Apr 5.

PMID:
29961509
7.

Congenital Titinopathy: Comprehensive characterization and pathogenic insights.

Oates EC, Jones KJ, Donkervoort S, Charlton A, Brammah S, Smith JE 3rd, Ware JS, Yau KS, Swanson LC, Whiffin N, Peduto AJ, Bournazos A, Waddell LB, Farrar MA, Sampaio HA, Teoh HL, Lamont PJ, Mowat D, Fitzsimons RB, Corbett AJ, Ryan MM, O'Grady GL, Sandaradura SA, Ghaoui R, Joshi H, Marshall JL, Nolan MA, Kaur S, Punetha J, Töpf A, Harris E, Bakshi M, Genetti CA, Marttila M, Werlauff U, Streichenberger N, Pestronk A, Mazanti I, Pinner JR, Vuillerot C, Grosmann C, Camacho A, Mohassel P, Leach ME, Foley AR, Bharucha-Goebel D, Collins J, Connolly AM, Gilbreath HR, Iannaccone ST, Castro D, Cummings BB, Webster RI, Lazaro L, Vissing J, Coppens S, Deconinck N, Luk HM, Thomas NH, Foulds NC, Illingworth MA, Ellard S, McLean CA, Phadke R, Ravenscroft G, Witting N, Hackman P, Richard I, Cooper ST, Kamsteeg EJ, Hoffman EP, Bushby K, Straub V, Udd B, Ferreiro A, North KN, Clarke NF, Lek M, Beggs AH, Bönnemann CG, MacArthur DG, Granzier H, Davis MR, Laing NG.

Ann Neurol. 2018 Jun;83(6):1105-1124. doi: 10.1002/ana.25241.

PMID:
29691892
8.

Dejerine-Sottas disease in childhood-Genetic and sonographic heterogeneity.

Hobbelink SMR, Brockley CR, Kennedy RA, Carroll K, de Valle K, Rao P, Davis MR, Laing NG, Voermans NC, Ryan MM, Yiu EM.

Brain Behav. 2018 Feb 21;8(4):e00919. doi: 10.1002/brb3.919. eCollection 2018 Apr.

9.

Unique clinical and neurophysiologic profile of a cohort of children with CMTX3.

Kanhangad M, Cornett K, Brewer MH, Nicholson GA, Ryan MM, Smith RL, Subramanian GM, Young HK, Züchner S, Kennerson ML, Burns J, Menezes MP.

Neurology. 2018 May 8;90(19):e1706-e1710. doi: 10.1212/WNL.0000000000005479. Epub 2018 Apr 6.

PMID:
29626178
10.

Gait and footwear in children and adolescents with Charcot-Marie-Tooth disease: A cross-sectional, case-controlled study.

Kennedy RA, McGinley JL, Paterson KL, Ryan MM, Carroll K.

Gait Posture. 2018 May;62:262-267. doi: 10.1016/j.gaitpost.2018.03.029. Epub 2018 Mar 19.

PMID:
29579702
11.

Investigation of the activation of the temporalis and masseter muscles in voluntary and spontaneous smile production.

Steele JE, Woodcock IR, Murphy AD, Ryan MM, Penington TJ, Coombs CJ.

J Plast Reconstr Aesthet Surg. 2018 Jul;71(7):1051-1057. doi: 10.1016/j.bjps.2018.02.013. Epub 2018 Mar 6.

PMID:
29555150
12.

Nusinersen for SMA: expanded access programme.

Farrar MA, Teoh HL, Carey KA, Cairns A, Forbes R, Herbert K, Holland S, Jones KJ, Menezes MP, Morrison M, Munro K, Villano D, Webster R, Woodcock IR, Yiu EM, Sampaio H, Ryan MM.

J Neurol Neurosurg Psychiatry. 2018 Sep;89(9):937-942. doi: 10.1136/jnnp-2017-317412. Epub 2018 Mar 16.

PMID:
29549190
13.

Utilising a multi-item questionnaire to assess household food security in Australia.

Butcher LM, O'Sullivan TA, Ryan MM, Lo J, Devine A.

Health Promot J Austr. 2018 Mar 15. doi: 10.1002/hpja.61. [Epub ahead of print]

PMID:
29543402
14.

A multinational study on motor function in early-onset FSHD.

Mah JK, Feng J, Jacobs MB, Duong T, Carroll K, de Valle K, Carty CL, Morgenroth LP, Guglieri M, Ryan MM, Clemens PR, Thangarajh M, Webster R, Smith E, Connolly AM, McDonald CM, Karachunski P, Tulinius M, Harper A, Cnaan A, Chen YW; Cooperative International Neuromuscular Research Group (CINRG) Investigators.

Neurology. 2018 Apr 10;90(15):e1333-e1338. doi: 10.1212/WNL.0000000000005297. Epub 2018 Mar 14.

PMID:
29540582
15.

DCC Is Required for the Development of Nociceptive Topognosis in Mice and Humans.

da Silva RV, Johannssen HC, Wyss MT, Roome RB, Bourojeni FB, Stifani N, Marsh APL, Ryan MM, Lockhart PJ, Leventer RJ, Richards LJ, Rosenblatt B, Srour M, Weber B, Zeilhofer HU, Kania A.

Cell Rep. 2018 Jan 30;22(5):1105-1114. doi: 10.1016/j.celrep.2018.01.004.

16.

Therapeutic Options to Improve Bone Health Outcomes in Duchenne Muscular Dystrophy: Zoledronic Acid and Pubertal Induction.

Lim A, Zacharin M, Pitkin J, de Valle K, Ryan MM, Simm PJ.

J Paediatr Child Health. 2017 Dec;53(12):1247-1248. doi: 10.1111/jpc.13692. No abstract available.

PMID:
29205648
17.

Acute flaccid myelitis in childhood: a retrospective cohort study.

Andersen EW, Kornberg AJ, Freeman JL, Leventer RJ, Ryan MM.

Eur J Neurol. 2017 Aug;24(8):1077-1083. doi: 10.1111/ene.13345. Epub 2017 Jun 22.

PMID:
28639345
18.

MCM3AP in recessive Charcot-Marie-Tooth neuropathy and mild intellectual disability.

Ylikallio E, Woldegebriel R, Tumiati M, Isohanni P, Ryan MM, Stark Z, Walsh M, Sawyer SL, Bell KM, Oshlack A, Lockhart PJ, Shcherbii M, Estrada-Cuzcano A, Atkinson D, Hartley T, Tetreault M, Cuppen I, van der Pol WL, Candayan A, Battaloglu E, Parman Y, van Gassen KLI, van den Boogaard MH, Boycott KM, Kauppi L, Jordanova A, Lönnqvist T, Tyynismaa H.

Brain. 2017 Aug 1;140(8):2093-2103. doi: 10.1093/brain/awx138.

PMID:
28633435
19.

Adverse Events of Atomoxetine in a Double-Blind Placebo-Controlled Study in Children with Autism.

Tumuluru RV, Corbett-Dick P, Aman MG, Smith T, Arnold LE, Pan X, Buchan-Page KA, Brown NV, Ryan MM, Hyman SL, Hellings J, Williams C, Hollway JA, Lecavalier L, Rice RR Jr, McAuliffe-Bellin S, Handen BL.

J Child Adolesc Psychopharmacol. 2017 Oct;27(8):708-714. doi: 10.1089/cap.2016.0187. Epub 2017 May 16.

20.

Deterioration in gait and functional ambulation in children and adolescents with Charcot-Marie-Tooth disease over 12 months.

Kennedy R, Carroll K, Paterson KL, Ryan MM, McGinley JL.

Neuromuscul Disord. 2017 Jul;27(7):658-666. doi: 10.1016/j.nmd.2017.04.005. Epub 2017 Apr 19.

PMID:
28495045
21.

Diagnostic and cost utility of whole exome sequencing in peripheral neuropathy.

Walsh M, Bell KM, Chong B, Creed E, Brett GR, Pope K, Thorne NP, Sadedin S, Georgeson P, Phelan DG, Day T, Taylor JA, Sexton A, Lockhart PJ, Kiers L, Fahey M, Macciocca I, Gaff CL, Oshlack A, Yiu EM, James PA, Stark Z, Ryan MM; Melbourne Genomics Health Alliance.

Ann Clin Transl Neurol. 2017 Apr 26;4(5):318-325. doi: 10.1002/acn3.409. eCollection 2017 May.

22.

Can in-the-moment diary methods measure health-related quality of life in Duchenne muscular dystrophy?

Bray P, Bundy AC, Ryan MM, North KN.

Qual Life Res. 2017 May;26(5):1145-1152. doi: 10.1007/s11136-016-1442-z. Epub 2016 Nov 3.

PMID:
27812849
23.

A prospective evaluation of whole-exome sequencing as a first-tier molecular test in infants with suspected monogenic disorders.

Stark Z, Tan TY, Chong B, Brett GR, Yap P, Walsh M, Yeung A, Peters H, Mordaunt D, Cowie S, Amor DJ, Savarirayan R, McGillivray G, Downie L, Ekert PG, Theda C, James PA, Yaplito-Lee J, Ryan MM, Leventer RJ, Creed E, Macciocca I, Bell KM, Oshlack A, Sadedin S, Georgeson P, Anderson C, Thorne N, Melbourne Genomics Health Alliance, Gaff C, White SM.

Genet Med. 2016 Nov;18(11):1090-1096. doi: 10.1038/gim.2016.1. Epub 2016 Mar 3.

PMID:
26938784
24.

An In Vitro Perfusion System to Enhance Outflow Studies in Mouse Eyes.

Kizhatil K, Chlebowski A, Tolman NG, Freeburg NF, Ryan MM, Shaw NN, Kokini AD, Marchant JK, John SW.

Invest Ophthalmol Vis Sci. 2016 Oct 1;57(13):5207-5215. doi: 10.1167/iovs.16-19481.

25.

Fifty years of paediatric neurology in Australasia.

Shield LK, Riney K, Antony JH, Ouvrier RA, Ryan MM.

J Paediatr Child Health. 2016 Sep;52(9):861-4. doi: 10.1111/jpc.13286. No abstract available.

PMID:
27650142
26.

Loss of function of SLC25A46 causes lethal congenital pontocerebellar hypoplasia.

Wan J, Steffen J, Yourshaw M, Mamsa H, Andersen E, Rudnik-Schöneborn S, Pope K, Howell KB, McLean CA, Kornberg AJ, Joseph J, Lockhart PJ, Zerres K, Ryan MM, Nelson SF, Koehler CM, Jen JC.

Brain. 2016 Nov 1;139(11):2877-2890. doi: 10.1093/brain/aww212.

27.

Neurophysiological profile of peripheral neuropathy associated with childhood mitochondrial disease.

Menezes MP, Rahman S, Bhattacharya K, Clark D, Christodoulou J, Ellaway C, Farrar M, Pitt M, Sampaio H, Ware TL, Wedatilake Y, Thorburn DR, Ryan MM, Ouvrier R.

Mitochondrion. 2016 Sep;30:162-7. doi: 10.1016/j.mito.2016.07.014. Epub 2016 Jul 27.

PMID:
27475922
28.

Whole Genome Sequencing Identifies a 78 kb Insertion from Chromosome 8 as the Cause of Charcot-Marie-Tooth Neuropathy CMTX3.

Brewer MH, Chaudhry R, Qi J, Kidambi A, Drew AP, Menezes MP, Ryan MM, Farrar MA, Mowat D, Subramanian GM, Young HK, Zuchner S, Reddel SW, Nicholson GA, Kennerson ML.

PLoS Genet. 2016 Jul 20;12(7):e1006177. doi: 10.1371/journal.pgen.1006177. eCollection 2016 Jul.

29.

Pathogenic mechanisms underlying X-linked Charcot-Marie-Tooth neuropathy (CMTX6) in patients with a pyruvate dehydrogenase kinase 3 mutation.

Perez-Siles G, Ly C, Grant A, Drew AP, Yiu EM, Ryan MM, Chuang DT, Tso SC, Nicholson GA, Kennerson ML.

Neurobiol Dis. 2016 Oct;94:237-44. doi: 10.1016/j.nbd.2016.07.001. Epub 2016 Jul 5.

PMID:
27388934
30.

Cerebral palsy is not a diagnosis: A case report of a novel atlastin-1 mutation.

Andersen EW, Leventer RJ, Reddihough DS, Davis MR, Ryan MM.

J Paediatr Child Health. 2016 Jun;52(6):669-71. doi: 10.1111/jpc.13200. No abstract available.

PMID:
27333849
31.

Describing nutrition in spinal muscular atrophy: A systematic review.

Moore GE, Lindenmayer AW, McConchie GA, Ryan MM, Davidson ZE.

Neuromuscul Disord. 2016 Jul;26(7):395-404. doi: 10.1016/j.nmd.2016.05.005. Epub 2016 May 10. Review.

PMID:
27241822
32.

Clinical practice considerations in facioscapulohumeral muscular dystrophy Sydney, Australia, 21 September 2015.

Tawil R, Mah JK, Baker S, Wagner KR, Ryan MM; Sydney Workshop Participants.

Neuromuscul Disord. 2016 Jul;26(7):462-71. doi: 10.1016/j.nmd.2016.03.007. Epub 2016 Apr 25. No abstract available.

PMID:
27185458
33.

Complete callosal agenesis, pontocerebellar hypoplasia, and axonal neuropathy due to AMPD2 loss.

Marsh AP, Lukic V, Pope K, Bromhead C, Tankard R, Ryan MM, Yiu EM, Sim JC, Delatycki MB, Amor DJ, McGillivray G, Sherr EH, Bahlo M, Leventer RJ, Lockhart PJ.

Neurol Genet. 2015 Jul 16;1(2):e16. doi: 10.1212/NXG.0000000000000014. eCollection 2015 Aug.

34.

Authors' Response to Commentary.

de Valle KL, Davidson ZE, Kennedy RA, Ryan MM, Carroll KM.

J Pediatr Rehabil Med. 2016;9(1):77. doi: 10.3233/PRM-160365. No abstract available.

PMID:
26966804
35.

Physical activity and the use of standard and complementary therapies in Duchenne and Becker muscular dystrophies.

de Valle KL, Davidson ZE, Kennedy RA, Ryan MM, Carroll KM.

J Pediatr Rehabil Med. 2016;9(1):55-63. doi: 10.3233/PRM-160364.

PMID:
26966801
36.

Urinary Soluble CD163 in Active Renal Vasculitis.

O'Reilly VP, Wong L, Kennedy C, Elliot LA, O'Meachair S, Coughlan AM, O'Brien EC, Ryan MM, Sandoval D, Connolly E, Dekkema GJ, Lau J, Abdulahad WH, Sanders JS, Heeringa P, Buckley C, O'Brien C, Finn S, Cohen CD, Lindemeyer MT, Hickey FB, O'Hara PV, Feighery C, Moran SM, Mellotte G, Clarkson MR, Dorman AJ, Murray PT, Little MA.

J Am Soc Nephrol. 2016 Sep;27(9):2906-16. doi: 10.1681/ASN.2015050511. Epub 2016 Mar 3. Erratum in: J Am Soc Nephrol. 2018 Aug;29(8):2255.

37.

EPG5-related Vici syndrome: a paradigm of neurodevelopmental disorders with defective autophagy.

Byrne S, Jansen L, U-King-Im JM, Siddiqui A, Lidov HG, Bodi I, Smith L, Mein R, Cullup T, Dionisi-Vici C, Al-Gazali L, Al-Owain M, Bruwer Z, Al Thihli K, El-Garhy R, Flanigan KM, Manickam K, Zmuda E, Banks W, Gershoni-Baruch R, Mandel H, Dagan E, Raas-Rothschild A, Barash H, Filloux F, Creel D, Harris M, Hamosh A, Kölker S, Ebrahimi-Fakhari D, Hoffmann GF, Manchester D, Boyer PJ, Manzur AY, Lourenco CM, Pilz DT, Kamath A, Prabhakar P, Rao VK, Rogers RC, Ryan MM, Brown NJ, McLean CA, Said E, Schara U, Stein A, Sewry C, Travan L, Wijburg FA, Zenker M, Mohammed S, Fanto M, Gautel M, Jungbluth H.

Brain. 2016 Mar;139(Pt 3):765-81. doi: 10.1093/brain/awv393.

38.

Neurologic Melioidosis: Case Report of a Rare Cause of Acute Flaccid Paralysis.

Andersen EW, Mackay MT, Ryan MM.

J Pediatr. 2016 Mar;170:319-21. doi: 10.1016/j.jpeds.2015.12.003. Epub 2016 Jan 8.

PMID:
26778096
39.

Next generation sequencing in a large cohort of patients presenting with neuromuscular disease before or at birth.

Todd EJ, Yau KS, Ong R, Slee J, McGillivray G, Barnett CP, Haliloglu G, Talim B, Akcoren Z, Kariminejad A, Cairns A, Clarke NF, Freckmann ML, Romero NB, Williams D, Sewry CA, Colley A, Ryan MM, Kiraly-Borri C, Sivadorai P, Allcock RJ, Beeson D, Maxwell S, Davis MR, Laing NG, Ravenscroft G.

Orphanet J Rare Dis. 2015 Nov 17;10:148. doi: 10.1186/s13023-015-0364-0.

40.

Atomoxetine, Parent Training, and Their Combination in Children With Autism Spectrum Disorder and Attention-Deficit/Hyperactivity Disorder.

Handen BL, Aman MG, Arnold LE, Hyman SL, Tumuluru RV, Lecavalier L, Corbett-Dick P, Pan X, Hollway JA, Buchan-Page KA, Silverman LB, Brown NV, Rice RR Jr, Hellings J, Mruzek DW, McAuliffe-Bellin S, Hurt EA, Ryan MM, Levato L, Smith T.

J Am Acad Child Adolesc Psychiatry. 2015 Nov;54(11):905-15. doi: 10.1016/j.jaac.2015.08.013. Epub 2015 Sep 3.

41.

'A short time but a lovely little short time': Bereaved parents' experiences of having a child with spinal muscular atrophy type 1.

Higgs EJ, McClaren BJ, Sahhar MA, Ryan MM, Forbes R.

J Paediatr Child Health. 2016 Jan;52(1):40-6. doi: 10.1111/jpc.12993. Epub 2015 Sep 10.

PMID:
26437687
42.

Dynamical properties of gene regulatory networks involved in long-term potentiation.

Nido GS, Ryan MM, Benuskova L, Williams JM.

Front Mol Neurosci. 2015 Aug 7;8:42. doi: 10.3389/fnmol.2015.00042. eCollection 2015.

43.

Nemaline Myopathy.

North KN, Ryan MM.

In: Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJH, Stephens K, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2018.
2002 Jun 19 [updated 2015 Jun 11].

44.

Unraveling the pathogenesis of ARX polyalanine tract variants using a clinical and molecular interfacing approach.

Marques I, Sá MJ, Soares G, Mota Mdo C, Pinheiro C, Aguiar L, Amado M, Soares C, Calado A, Dias P, Sousa AB, Fortuna AM, Santos R, Howell KB, Ryan MM, Leventer RJ, Sachdev R, Catford R, Friend K, Mattiske TR, Shoubridge C, Jorge P.

Mol Genet Genomic Med. 2015 May;3(3):203-14. doi: 10.1002/mgg3.133. Epub 2015 Feb 25.

45.

An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels.

Yiu EM, Tai G, Peverill RE, Lee KJ, Croft KD, Mori TA, Scheiber-Mojdehkar B, Sturm B, Praschberger M, Vogel AP, Rance G, Stephenson SE, Sarsero JP, Stockley C, Lee CY, Churchyard A, Evans-Galea MV, Ryan MM, Lockhart PJ, Corben LA, Delatycki MB.

J Neurol. 2015 May;262(5):1344-53. doi: 10.1007/s00415-015-7719-2. Epub 2015 Apr 7.

PMID:
25845763
46.

Aging alters long-term potentiation--related gene networks and impairs synaptic protein synthesis in the rat hippocampus.

Ryan MM, Guévremont D, Luxmanan C, Abraham WC, Williams JM.

Neurobiol Aging. 2015 May;36(5):1868-80. doi: 10.1016/j.neurobiolaging.2015.01.012. Epub 2015 Jan 19.

PMID:
25716081
47.

A diagnostic approach to recurrent myalgia and rhabdomyolysis in children.

Chan EK, Kornberg AJ, Ryan MM.

Arch Dis Child. 2015 Aug;100(8):793-7. doi: 10.1136/archdischild-2014-307663. Epub 2015 Jan 29. Review.

PMID:
25633066
48.

A mixed methods study of age at diagnosis and diagnostic odyssey for Duchenne muscular dystrophy.

Wong SH, McClaren BJ, Archibald AD, Weeks A, Langmaid T, Ryan MM, Kornberg A, Metcalfe SA.

Eur J Hum Genet. 2015 Oct;23(10):1294-300. doi: 10.1038/ejhg.2014.301. Epub 2015 Jan 28.

49.

Peripheral nerve ultrasound in pediatric Charcot-Marie-Tooth disease type 1A.

Yiu EM, Brockley CR, Lee KJ, Carroll K, de Valle K, Kennedy R, Rao P, Delatycki MB, Ryan MM.

Neurology. 2015 Feb 10;84(6):569-74. doi: 10.1212/WNL.0000000000001236. Epub 2015 Jan 9.

PMID:
25576636
50.

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.

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