Tag «Duchenne muscular dystrophy»

SYN-01

It’s only fair to share…     SYN-01, SYN-510 Synthena AG Preclinical Synthena , presumed to be under license from  University of Bern , is investigating (presumably SYN-01 ), a lead from the tricyclo(tc)-DNA based antisense oligonucleotides (AON) developed using its proprietary tricyclo-DNA technology platform, for the treatment of Duchenne muscular dystrophy. In January 2017, …

FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

It’s only fair to share… CAS 1173755-55-9 eteplirsen, eteplirsén [Spanish], étéplirsen [French] , eteplirsenum [Latin], этеплирсен [Russian], إيتيبليرسان [Arabic] Structure credit http://lgmpharma.com/eteplirsen-still-proves-efficacious-duchenne-drug/ FDA grants accelerated approval to first drug for Duchenne muscular dystrophy New therapy addresses unmet medical need The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy …