1
|
Unguru Y: Ethical challenges in
early-phase pediatric research for life-limiting illness. Semin
Pediatr Neurol. 22:177–186. 2015.PubMed/NCBI View Article : Google Scholar
|
2
|
Constantin B: Dystrophin complex functions
as a scaffold for signalling proteins. Biochim Biophys Acta.
1838:635–642. 2014.PubMed/NCBI View Article : Google Scholar
|
3
|
McDonald CM, Henricson EK, Abresch RT,
Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP and Cnaan A:
Long-term effects of glucocorticoids on function, quality of life,
and survival in patients with Duchenne muscular dystrophy: A
prospective cohort study. Lancet. 391:451–461. 2018.PubMed/NCBI View Article : Google Scholar
|
4
|
Archer JD, Vargas CC and Anderson JE:
Persistent and improved functional gain in mdx dystrophic mice
after treatment with L-arginine and deflazacort. FASEB J.
20:738–740. 2006.PubMed/NCBI View Article : Google Scholar
|
5
|
Hörster I, Weigt-Usinger K, Carmann C,
Chobanyan-Jürgens K, Köhler C, Schara U, Kayacelebi AA, Beckmann B,
Tsikas D and Lücke T: The L-arginine/NO pathway and homoarginine
are altered in Duchenne muscular dystrophy and improved by
glucocorticoids. Amino Acids. 47:1853–1863. 2015.PubMed/NCBI View Article : Google Scholar
|
6
|
Vianello S, Pantic B, Fusto A, Bello L,
Galletta E, Borgia D, Gavassini BF, Semplicini C, Sorarù G,
Vitiello L and Pegoraro E: SPP1 genotype and glucocorticoid
treatment modify osteopontin expression in Duchenne muscular
dystrophy cells. Hum Mol Genet. 26:3342–3351. 2017.PubMed/NCBI View Article : Google Scholar
|
7
|
McRae N, Forgan L, McNeill B, Addinsall A,
McCulloch D, Van der Poel C and Stupka N: Glucocorticoids improve
myogenic differentiation in vitro by suppressing the synthesis of
versican, a transitional matrix protein overexpressed in dystrophic
skeletal muscles. Int J Mol Sci. 18(2629)2017.PubMed/NCBI View Article : Google Scholar
|
8
|
Morrison-Nozik A, Anand P, Zhu H, Duan Q,
Sabeh M, Prosdocimo DA, Lemieux ME, Nordsborg N, Russell AP, MacRae
CA, et al: Glucocorticoids enhance muscle endurance and ameliorate
Duchenne muscular dystrophy through a defined metabolic program.
Proc Natl Acad Sci USA. 112:E6780–E6789. 2015.PubMed/NCBI View Article : Google Scholar
|
9
|
Manzur AY, Kuntzer T, Pike M and Swan A:
Glucocorticoid corticosteroids for Duchenne muscular dystrophy.
Cochrane Database Syst Rev: Jan 23, 2008. doi:
org/10.1002/14651858.CD003725.pub3.
|
10
|
Baltgalvis KA, Call JA, Nikas JB and Lowe
DA: Effects of prednisolone on skeletal muscle contractility in mdx
mice. Muscle Nerve. 40:443–454. 2009.PubMed/NCBI View Article : Google Scholar
|
11
|
Jensen L, Petersson SJ, Illum NO,
Laugaard-Jacobsen HC, Thelle T, Jørgensen LH and Schrøder HD:
Muscular response to the first three months of deflazacort
treatment in boys with Duchenne muscular dystrophy. J Musculoskelet
Neuronal Interact. 17:8–18. 2017.PubMed/NCBI
|
12
|
Merlini L, Gennari M, Malaspina E, Cecconi
I, Armaroli A, Gnudi S, Talim B, Ferlini A, Cicognani A and
Franzoni E: Early corticosteroid treatment in 4 Duchenne muscular
dystrophy patients: 14-year follow-up. Muscle Nerve. 45:796–802.
2012.PubMed/NCBI View Article : Google Scholar
|
13
|
Birnkrant DJ, Bushby K, Bann CM, Apkon SD,
Blackwell A, Colvin MK, Cripe L, Herron AR, Kennedy A, Kinnett K,
et al: Diagnosis and management of Duchenne muscular dystrophy,
part 3: Primary care, emergency management, psychosocial care, and
transitions of care across the lifespan. Lancet Neurol. 17:445–455.
2018.PubMed/NCBI View Article : Google Scholar
|
14
|
Tandon A, Villa CR, Hor KN, Jefferies JL,
Gao Z, Towbin JA, Wong BL, Mazur W, Fleck RJ, Sticka JJ, et al:
Myocardial fibrosis burden predicts left ventricular ejection
fraction and is associated with age and steroid treatment duration
in Duchenne muscular dystrophy. J Am Heart Assoc.
4(e001338)2015.PubMed/NCBI View Article : Google Scholar
|
15
|
Sawnani H, Horn PS, Wong B, Darmahkasih A,
Rybalsky I, Shellenbarger KC, Tian C, Rutter MM, Simakajornboon N,
Amin R, et al: Comparison of pulmonary function decline in
steroid-treated and steroid-naïve patients with Duchenne muscular
dystrophy. J Pediatr. 210:194–200.e2. 2019.PubMed/NCBI View Article : Google Scholar
|
16
|
Annexstad EJ, Fagerheim T, Holm I and
Rasmussen M: Molecular and clinical characteristics of a national
cohort of paediatric Duchenne muscular dystrophy patients in
Norway. J Neuromuscul Dis. 6:349–359. 2019.PubMed/NCBI View Article : Google Scholar
|
17
|
Qi B, Jing HU, Zhe Zhao, Shen Hong Rui and
Na LI: Clinical analysis of 155 patients with Duchenne muscular
dystrophy. Chin J Contemp Neurol Neurosurg. 15:380–386. 2015.
|
18
|
Hu J, Ye Y, Kong M, Hong S, Cheng L, Wang
Q, Qin J, Zou L and Jiang L: Daily prednisone treatment in Duchenne
muscular dystrophy in southwest China. Muscle Nerve. 52:1001–1007.
2015.PubMed/NCBI View Article : Google Scholar
|
19
|
Wong BL, Rybalsky I, Shellenbarger KC,
Tian C, McMahon MA, Rutter MM, Sawnani H and Jefferies JL:
Long-term outcome of interdisciplinary management of patients with
Duchenne muscular dystrophy receiving daily glucocorticoid
treatment. J Pediatr. 182:296–303.e1. 2017.PubMed/NCBI View Article : Google Scholar
|
20
|
Sato Y, Yamauchi A, Urano M, Kondo E and
Saito K: Corticosteroid therapy for Duchenne muscular dystrophy:
Improvement of psychomotor function. Pediatr Neurol. 50:31–37.
2014.PubMed/NCBI View Article : Google Scholar
|
21
|
Ishigaki K, Kato I, Murakami T, Saito T,
Sato T, Kajino S, Nakanishi T and Osawa M: Long-term and low-dose
steroid therapy for cardiomyopathy in Duchenne muscular dystrophy
patients. J Tokyo Women's Med Coll. 83:E14–E19. 2013.
|
22
|
Connolly AM, Zaidman CM, Golumbek PT,
Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM,
Iannaccone ST, Anand P, et al: Twice-weekly glucocorticosteroids in
infants and young boys with Duchenne muscular dystrophy. Muscle
Nerve. 59:650–657. 2019.PubMed/NCBI View Article : Google Scholar
|
23
|
Escolar DM, Hache LP, Clemens PR, Cnaan A,
McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y,
Lotze T, et al: Randomized, blinded trial of weekend vs. daily
prednisone in Duchenne muscular dystrophy. Neurology. 77:444–452.
2011.PubMed/NCBI View Article : Google Scholar
|
24
|
Crabtree NJ, Adams JE, Padidela R, Shaw
NJ, Högler W, Roper H, Hughes I, Daniel A and Mughal MZ: Growth,
bone health and ambulatory status of boys with DMD treated with
daily vs. intermittent oral glucocorticoid regimen. Bone.
116:181–186. 2018.PubMed/NCBI View Article : Google Scholar
|
25
|
Abutaleb AR, McNally EM, Khan SS, Anderson
AS, Carr JC and Wilcox JE: Myocarditis in Duchenne muscular
dystrophy after changing steroids. JAMA Cardiol. 3:1006–1010.
2018.PubMed/NCBI View Article : Google Scholar
|
26
|
Griggs RC, Miller JP, Greenberg CR,
Fehlings DL, Pestronk A, Mendell JR, Moxley RT III, King W, Kissel
JT, Cwik V, et al: Efficacy and safety of deflazacort vs prednisone
and placebo for Duchenne muscular dystrophy. Neurology.
87:2123–2131. 2016.PubMed/NCBI View Article : Google Scholar
|
27
|
Matthews E, Brassington R, Kuntzer T,
Jichi F and Manzur AY: Corticosteroids for the treatment of
Duchenne muscular dystrophy. Cochrane Database Syst Rev.
(CD003725)2016.PubMed/NCBI View Article : Google Scholar
|
28
|
McDonald CM, Gordish-Dressman H, Henricson
EK, Duong T, Joyce NC, Jhawar S, Leinonen M, Hsu F, Connolly AM,
Cnaan A, et al: Longitudinal pulmonary function testing outcome
measures in Duchenne muscular dystrophy: Long-term natural history
with and without glucocorticoids. Neuromuscul Disord. 28:897–909.
2018.PubMed/NCBI View Article : Google Scholar
|
29
|
Pane M, Fanelli L, Mazzone ES, Olivieri G,
D'Amico A, Messina S, Scutifero M, Battini R, Petillo R, Frosini S,
et al: Benefits of glucocorticoids in non-ambulant boys/men with
Duchenne muscular dystrophy: A multicentric longitudinal study
using the performance of upper limb test. Neuromuscul Disord.
25:749–753. 2015.PubMed/NCBI View Article : Google Scholar
|
30
|
Karimzadeh P and Ghazavi A: Comparison of
deflazacort and prednisone in Duchenne muscular dystrophy. Iran J
Child Neurol. 6:5–12. 2012.
|
31
|
Moxley RT III, Pandya S, Ciafaloni E, Fox
DJ and Campbell K: Change in natural history of Duchenne muscular
dystrophy with long-term corticosteroid treatment: Implications for
management. J Child Neurol. 25:1116–1129. 2010.PubMed/NCBI View Article : Google Scholar
|
32
|
Matsumura T, Saito T, Fujimura H, Shinno S
and Sakoda S: A longitudinal cause-of-death analysis of patients
with Duchenne muscular dystrophy. Rinsho Shinkeigaku. 51:743–750.
2011.PubMed/NCBI View Article : Google Scholar : (In Japanese).
|
33
|
Al-Zougbi A, Mathews KD and Shibli-Rahhal
A: Use of bone age for evaluating bone density in patients with
Duchenne muscular dystrophy: A preliminary report. Muscle Nerve.
59:422–425. 2019.PubMed/NCBI View Article : Google Scholar
|
34
|
Ma J, McMillan HJ, Karagüzel G, Goodin C,
Wasson J, Matzinger MA, DesClouds P, Cram D, Page M, Konji VN, et
al: The time to and determinants of first fractures in boys with
Duchenne muscular dystrophy. Osteoporos Int. 28:597–608.
2017.PubMed/NCBI View Article : Google Scholar
|
35
|
Lim KR, Maruyama R and Yokota T:
Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug
Des Devel Ther. 11:533–545. 2017.PubMed/NCBI View Article : Google Scholar
|
36
|
Conklin LS, Damsker JM, Hoffman EP, Jusko
WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK,
Guglieri M, et al: Phase IIa trial in Duchenne muscular dystrophy
shows vamorolone is a first-in-class dissociative steroidal
anti-inflammatory drug. Pharmacol Res. 136:140–150. 2018.PubMed/NCBI View Article : Google Scholar
|
37
|
Kumaki D, Nakamura Y, Sakai N, Kosho T,
Nakamura A, Hirabayashi S, Suzuki T, Kamimura M and Kato H:
Efficacy of denosumab for glucocorticoid-induced osteoporosis in an
adolescent patient with Duchenne muscular dystrophy: A case report.
JBJS Case Connect. 8(e22)2018.PubMed/NCBI View Article : Google Scholar
|
38
|
Nagy S, Hafner P, Schmidt S, Rubino-Nacht
D, Schädelin S, Bieri O and Fischer D: Tamoxifen in Duchenne
muscular dystrophy (TAMDMD): Study protocol for a multicenter,
randomized, placebo-controlled, double-blind phase 3 trial. Trials.
20(637)2019.PubMed/NCBI View Article : Google Scholar
|
39
|
Buyse GM, Voit T, Schara U, Straathof CS,
D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald
CM, et al: Efficacy of idebenone on respiratory function in
patients with Duchenne muscular dystrophy not using glucocorticoids
(DELOS): A double-blind randomised placebo-controlled phase 3
trial. Lancet. 385:1748–1757. 2015.PubMed/NCBI View Article : Google Scholar
|
40
|
Victor RG, Sweeney HL, Finkel R, McDonald
CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL,
Topaloglu H, et al: A phase 3 randomized placebo-controlled trial
of tadalafil for Duchenne muscular dystrophy. Neurology.
89:1811–1820. 2017.PubMed/NCBI View Article : Google Scholar
|
41
|
Wood CL, Cheetham TD, Guglieri M, Bushby
K, Owen C, Johnstone H and Straub V: Testosterone treatment of
pubertal delay in Duchenne muscular dystrophy. Neuropediatrics.
46:371–376. 2015.PubMed/NCBI View Article : Google Scholar
|
42
|
Perera N, Sampaio H, Woodhead H and Farrar
M: Fracture in Duchenne muscular dystrophy: Natural history and
vitamin D deficiency. J Child Neurol. 31:1181–1187. 2016.PubMed/NCBI View Article : Google Scholar
|
43
|
Srinivasan R, Rawlings D, Wood CL,
Cheetham T, Moreno AC, Mayhew A, Eagle M, Guglieri M, Straub V,
Owen C, et al: Prophylactic oral bisphosphonate therapy in duchenne
muscular dystrophy. Muscle Nerve. 54:79–85. 2016.PubMed/NCBI View Article : Google Scholar
|