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Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy.
Mendell JR, Proud C, Zaidman CM, Mason S, Darton E, Wang S, Wandel C, Murphy AP, Mercuri E, Muntoni F, McDonald CM. Mendell JR, et al. Pediatr Neurol. 2024 Apr;153:11-18. doi: 10.1016/j.pediatrneurol.2024.01.003. Epub 2024 Jan 5. Pediatr Neurol. 2024. PMID: 38306745 Free article.
BACKGROUND: Delandistrogene moxeparvovec is a gene transfer therapy approved in the United States, United Arab Emirates, and Qatar for the treatment of ambulatory patients aged four through five years with a confirmed Duchenne muscular dystrophy (DMD)-causing mutation in t …
BACKGROUND: Delandistrogene moxeparvovec is a gene transfer therapy approved in the United States, United Arab Emirates, and Qatar for the t …
Corticosteroids in Duchenne muscular dystrophy: a reappraisal.
Wong BL, Christopher C. Wong BL, et al. J Child Neurol. 2002 Mar;17(3):183-90. doi: 10.1177/088307380201700306. J Child Neurol. 2002. PMID: 12026233 Review.
Duchenne muscular dystrophy is the most common and most severe form of childhood muscular dystrophies, resulting in early loss of ambulation between the ages of 7 and 13 years and death in the teens and twenties. Despite the phenomenal advances made in the understanding of …
Duchenne muscular dystrophy is the most common and most severe form of childhood muscular dystrophies, resulting in early loss of amb …
Impact of three genetic musculoskeletal diseases: a comparative synthesis of achondroplasia, Duchenne muscular dystrophy and osteogenesis imperfecta.
Dogba MJ, Rauch F, Douglas E, Bedos C. Dogba MJ, et al. Health Qual Life Outcomes. 2014 Oct 25;12:151. doi: 10.1186/s12955-014-0151-y. Health Qual Life Outcomes. 2014. PMID: 25649344 Free PMC article. Review.
Achondroplasia, Duchenne muscular dystrophy, and osteogenesis imperfecta are among the most frequent rare genetic disorders affecting the musculoskeletal system in children. ...This literature review aims to classify, summarize and compare these non-medical impacts of acho …
Achondroplasia, Duchenne muscular dystrophy, and osteogenesis imperfecta are among the most frequent rare genetic disorders affecting …
Model organisms in the fight against muscular dystrophy: lessons from drosophila and Zebrafish.
Plantié E, Migocka-Patrzałek M, Daczewska M, Jagla K. Plantié E, et al. Molecules. 2015 Apr 9;20(4):6237-53. doi: 10.3390/molecules20046237. Molecules. 2015. PMID: 25859781 Free PMC article. Review.
More than 30 types of MD have been described so far; those most thoroughly studied are Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and congenital MDs. ...
More than 30 types of MD have been described so far; those most thoroughly studied are Duchenne muscular dystrophy (DMD), myotonic dy …
Left ventricular noncompaction in Duchenne muscular dystrophy.
Statile CJ, Taylor MD, Mazur W, Cripe LH, King E, Pratt J, Benson DW, Hor KN. Statile CJ, et al. J Cardiovasc Magn Reson. 2013 Aug 1;15(1):67. doi: 10.1186/1532-429X-15-67. J Cardiovasc Magn Reson. 2013. PMID: 23914774 Free PMC article.
The objective of this study is to define the prevalence of LVNC in the Duchenne Muscular Dystrophy (DMD) population and characterize its relationship to global LV function. ...
The objective of this study is to define the prevalence of LVNC in the Duchenne Muscular Dystrophy (DMD) population and characterize …
Myocardial Strain Using Cardiac MR Feature Tracking and Speckle Tracking Echocardiography in Duchenne Muscular Dystrophy Patients.
Siegel B, Olivieri L, Gordish-Dressman H, Spurney CF. Siegel B, et al. Pediatr Cardiol. 2018 Mar;39(3):478-483. doi: 10.1007/s00246-017-1777-4. Epub 2017 Nov 29. Pediatr Cardiol. 2018. PMID: 29188318 Free PMC article.
Duchenne muscular dystrophy (DMD) is an inherited X-linked disorder with an incidence of 1 in 3500 male births, and cardiomyopathy is becoming the leading cause of death. ...
Duchenne muscular dystrophy (DMD) is an inherited X-linked disorder with an incidence of 1 in 3500 male births, and cardiomyopathy is …
Copper-catalyzed direct C-H fluoroalkenylation of heteroarenes.
Rousée K, Schneider C, Bouillon JP, Levacher V, Hoarau C, Couve-Bonnaire S, Pannecoucke X. Rousée K, et al. Org Biomol Chem. 2016 Jan 7;14(1):353-7. doi: 10.1039/c5ob02213d. Epub 2015 Nov 25. Org Biomol Chem. 2016. PMID: 26603641
The synthesis of fluorinated analogues of biomolecules and therapeutic agents for the treatment of Duchenne muscular dystrophy as application is reported....
The synthesis of fluorinated analogues of biomolecules and therapeutic agents for the treatment of Duchenne muscular dystrophy as app …
[Functional and orthopaedic aspects of dystrophinopathies].
Boulay C, Finidori G. Boulay C, et al. Arch Pediatr. 2015 Dec;22(12 Suppl 1):12S42-50. doi: 10.1016/S0929-693X(16)30008-2. Arch Pediatr. 2015. PMID: 26773586 French.
Although the clinical picture and the natural progression of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) differ, borderline forms exist. ...
Although the clinical picture and the natural progression of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) di …
20 results