Tag «Duchenne muscular dystrophy»

IDEBENONE

It’s only fair to share… IDEBENONE 2-(10-Hydroxydecyl)-5,6-dimethoxy-3-methyl-1,4-benzoquinone Molecular FormulaC19H30O5 Average mass338.439 Da 58186-27-9 Idebenona, Idebenonum, CV 2619 Idesol KS-5193 Nemocebral SNT-MC17 идебенон إيديبينون 艾地苯醌 Puldysa (idebenone), for the treatment of Duchenne muscular dystrophy Title: Idebenone CAS Registry Number: 58186-27-9 CAS Name: 2-(10-Hydroxydecyl)-5,6-dimethoxy-3-methyl-2,5-cyclohexadiene-1,4-dione Additional Names: 6-(10-hydroxydecyl)-2,3-dimethoxy-5-methyl-1,4-benzoquinone; 2,3-dimethoxy-5-methyl-6-(10¢-hydroxydecyl)-1,4-benzoquinone; 6-(10-hydroxydecyl)ubiquinone Manufacturers’ Codes: CV-2619 Trademarks: Avan (Takeda); Daruma (Takeda); Lucebanol (Hormona); Mnesis (Takeda) Molecular Formula: C19H30O5 Molecular Weight: 338.44 Percent …

Golodirsen, ゴロジルセン;

It’s only fair to share…   Golodirsen RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2’a→5′)(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-G-m5U-m5U-C), 5′-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] Nucleic Acid Sequence Sequence Length: 25 Formula C305H481N138O112P25 CAS 1422959-91-8 Mol weight 8647.2841   Exon 53: NG-12-0163 Golodirsen SRP 4053 Nucleic Acid Sequence Sequence Length: 252 a 6 c 8 g 9 umodified FDA APPROVED, Vyondys 53, 019/12/12 Antisense oligonucleotide ゴロジルセン; Duchenne muscular dystrophy (DMD variant amenable …

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 …