Sarepta's treatment of DMD antisense RNA drug Exondys 51 (eteplirsen) 3 trials yield positive results
-
Last Update: 2020-06-09
-
Source: Internet
-
Author: User
Search more information of high quality chemicals, good prices and reliable suppliers, visit
www.echemi.com
Recently, a newly published study showed that Sarepta Therapeuticscompany(http://treats Duchenne's muscular dystrophy (DMD) antonymic rnadrug(http://Exo
ndirsen, all of which achieved positive decline in lung function in threetrial(http://of the post-validation studyExo
ndys 51 is the first innovative treatment approved by theFDA(http://for the treatment of specific DMD patients About Exo
ndys 51
Exo
ndys 51 is a sarepta-developed phosphamide dipolytosis oligonucleotide, targeted to the shear process of the anti-amyotrophic protein mRNA precursor, designed to introduce exon 51 jumps (exon 51 skipping) to produce short but functional antimuscular hymne protein According to statistics, about 13% of DMD patients carry genetic mutations suitable for exogenous 51 jump therapy Previous lying results have shown that Exo
ndys 51 reached several biological endpoints in clinical trials, including the proper completion of exon jumps at RNA production levels, the expression of antimyosphin, and increased muscle strength In the three post-market studies, Exo
ndys 51 can delay the decline of their lung function, regardless of the stage at which the patient develops
This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only.
This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of
the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed
description of the concern or complaint, to
service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content
will be removed immediately.
