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Duchenne muscular dystrophy

Disease ID:1210
Name:Duchenne muscular dystrophy
Associated with:0 target
2 immuno-relevant ligands
Description
A muscular dystrophy caused by mutations in the DMD gene found on the X chromosome, therefore males are affected whilst females can be carriers. Characterized by rapidly progressing muscle weakness and muscle atrophy initially involving the lower extremities and eventually affecting the whole body
Database Links
Disease Ontology: DOID:11723
OMIM: 19518854

Targets

No target related data available for Duchenne muscular dystrophy

Ligands

Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
deflazacort
Immuno Disease Comments: Deflazacort was approved by the US FDA in 2017 as a treatment for DMD.
Clinical Use: Deflazacort can be prescribed for many inflammatory conditions including asthma, rheumatoid arthritis, Crohn's disease, juvenile chronic arthritis, idiopathic thrombocytopenic purpura, polymyalgia rheumatica, systemic lupus erythematosus and ulcerative colitis. More recently approved to treat Duchenne muscular dystrophy [2]. | View clinical data
Bioactivity Comments: In vitro binding to rat kidney, thymus and liver glucocorticoid receptors is reported in [4]. | View biological activity
naproxcinod
Immuno Disease Comments: Naproxcinod has orphan drug designation for DMD in Europe and the US.
Clinical Use: Proof-of-concept in humans is reported in [3]. The EMA granted naproxcinod orphan designation for the treatment of Duchenne muscular dystrophy [5] in 2013, followed by the US FDA in 2015. Phase 3 studies comparing naproxcinod with naproxen or placebo in patients with osteoarthritis of the hip and knee have been completed [1,6]. | View clinical data
Bioactivity Comments: The biological activity of this drug will be determined by its cyclooxygenase inhibition () and -induced effects. See the separate ligand pages for each of these active components for further information. | View biological activity

References

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1. Baerwald C, Verdecchia P, Duquesroix B, Frayssinet H, Ferreira T. (2010) Efficacy, safety, and effects on blood pressure of naproxcinod 750 mg twice daily compared with placebo and naproxen 500 mg twice daily in patients with osteoarthritis of the hip: a randomized, double-blind, parallel-group, multicenter study. Arthritis Rheum., 62 (12): 3635-44. [PMID:20722026]

2. Griggs RC, Miller JP, Greenberg CR, Fehlings DL, Pestronk A, Mendell JR, Moxley 3rd RT, King W, Kissel JT, Cwik V et al.. (2016) Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology, 87 (20): 2123-2131. [PMID:27566742]

3. Hawkey CJ, Jones JI, Atherton CT, Skelly MM, Bebb JR, Fagerholm U, Jonzon B, Karlsson P, Bjarnason IT. (2003) Gastrointestinal safety of AZD3582, a cyclooxygenase inhibiting nitric oxide donator: proof of concept study in humans. Gut, 52 (11): 1537-42. [PMID:14570719]

4. Luzzani F, Glässer A. (1981) Differential binding in vitro to glucocorticoid receptors of deflazacort and prednisolone. Eur. J. Pharmacol., 76 (4): 427-30. [PMID:7327211]

5. Miglietta D, De Palma C, Sciorati C, Vergani B, Pisa V, Villa A, Ongini E, Clementi E. (2015) Naproxcinod shows significant advantages over naproxen in the mdx model of Duchenne Muscular Dystrophy. Orphanet J Rare Dis, 10: 101. [PMID:26296873]

6. Schnitzer TJ, Hochberg MC, Marrero CE, Duquesroix B, Frayssinet H, Beekman M. (2011) Efficacy and safety of naproxcinod in patients with osteoarthritis of the knee: a 53-week prospective randomized multicenter study. Semin. Arthritis Rheum., 40 (4): 285-97. [PMID:20828790]