FDA accelerates approval of Vyondys 53 for treatment of Duchenne muscular dystrophy
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Last Update: 2020-06-07
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Source: Internet
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Author: User
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today, the United StatesThe FDA's(http://announced the accelerated approval of Theof Sarepta Therapeuticscompany(http://of Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy (DMD) suitable for treatment with exon 53 skippingAbout Vyo
ndys 53
Vyo
ndys 53 is a phosphamide diamine developed by Sarepta? It targets the cutting process of the antiamatphinprotein mRNA precursor, introducing exon 53 jumps, designed to produce short but functional antimuscular honeting proteinThe efficacy of Vyo
ndys 53 was evaluated in a two-part clinical studyThe first part consisted of 12 DMD patients, 8 patients treated with Vyo
ndys 53, and 4 with placebo treatment The second part is the open label trial (http:// , including the first part of 12 patients and 13 new DMD patients In this study, after 48 weeks of treatment, the average amyotrophic protein level in patients increased from a normal level of 0.1% (baseline) to 1.02% of the normal level The accelerated approval of Vyo
ndys 53 is based on an alternative endpoint for elevated amyotrophin levels in the skeletal muscle of patients treated The FDA believes that elevated amyotrophic protein levels in the application may reasonably predict the clinical benefits of DMD patients Current clinical studies will assess whether Vyo
ndys 53 can improve motor function in Patients with DMD If clinical trials fail to prove clinical benefits, the FDA may withdraw approval for the drug (http://
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