Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics....
small molecule drugs such as aminoglycosides and negamycin. An oxadiazole, ataluren (previously PTC124), facilitates the selective read-through of aberrant...
currently available including gene therapy (Elevidys), and antisense drugs (Ataluren, Eteplirsen etc.). Other medications used include glucocorticoids (Deflazacort...
currently available including gene therapy (Elevidys), and antisense drugs (Ataluren, Eteplirsen etc.). Other medications used include glucocorticoids (Deflazacort...
the FDA in 2015. As of 2015, lumacaftor had no medical use on its own. Ataluren, targeting premature stop codons Kuk K, Taylor-Cousar JL (December 2015)...
approved drugs for human illness therapy is scarce. Ribavirin, branaplam, and ataluren are currently available medications that stabilize double-stranded RNA...
to dying muscle cells. These patients do not require antisense drugs (Ataluren, Eteplirsen etc.) as certain percentage of dystrophin is already expressed...
membrane stained biopsies at baseline and either 24 or 48 weeks. As of 2016, ataluren was the only approved drug in the EU to treat a specific subpopulation...
with PTC that produced ataluren—Sweeney was the last author on the initial publication of ataluren in Nature in 2007. Ataluren was approved to treat DMD...
tested in young boys with Duchenne muscular Dystrophy aged 6 to 11 years. Ataluren Biostrophin (experimental) Idebenone (experimental) Spreitzer H (26 May...
Nonsense mutations in the CDKL5 gene could be suppressed by compounds such as ataluren; (PTC Therapeutics), or similar next-generation translation stop readthrough...
100 times or more. In 2009, his group's mechanistic studies of PTC124 (Ataluren), then in clinical trials for subclasses of cystic fibrosis and muscular...
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