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Substance Name: Drisapersen sodium [USAN]
RN: 1181666-20-5
UNII: SDA7197G7Q
InChIKey: FZJHBHYSGPESDI-FWLJYILUSA-A

Note

  • ... is a synthetic, modified RNA molecule; Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations

Molecular Formula

  • C211-H256-N76-Na19-O119-P19-S19

Molecular Weight

  • 7395.3264
 

Classification Code

  • Treatment of Duchenne Muscular Dystrophy
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Names and Synonyms

Name of Substance

  • Drisapersen sodium [USAN]

Synonyms

  • All-P-ambo-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P-thiocytidylyl-(3'->5')-2'-O- methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P- thioguanylyl-(3'->5')-2'-O-methyl-P-thioguanylyl-(3'->5')-2'-O-methyl-P-thioadenylyl- (3'->5')-2'-O-methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thioguanylyl-(3'->5')-2'-O- methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P- thioguanylyl-(3'->5')-2'-O-methyl-P-thioguanylyl-(3'->5')-2'-O-methyl-P-thiocytidylyl- (3'->5')-2'-O-methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O- methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P- thiocytidylyl-(3'->5')-2'-O-methyluridine, nonadeca sodium
  • Drisapersen sodium
  • GSK2402968B
  • h51AON23 sodium salt
  • UNII-SDA7197G7Q

Systematic Name

  • RNA, (P-thio)(Um-Cm-Am-Am-Gm-Gm-Am-Am-Gm-Am-Um-Gm-Gm-Cm-Am-Um-Um- Um-Cm-Um), sodium salt (1:19)

Registry Numbers

CAS Registry Number

  • 1181666-20-5

FDA UNII

  • SDA7197G7Q

System Generated Number

  • 1181666205

Structure Descriptors

InChI

1S/C211H275N76O119P19S19.19Na/c1-329-136-116(295)77(369-178(136)271-33-24-99(289)252-206(271)304)40-349-407(310,426)389-118-79(371-180(138(118)331-3)268-30-21-96(212)249-203(268)301)42-351-409(312,428)392-121-81(373-183(141(121)334-6)273-35-26-101(291)254-208(273)306)44-352-410(313,429)393-122-82(374-184(142(122)335-7)274-36-27-102(292)255-209(274)307)45-353-411(314,430)394-123-83(375-185(143(123)336-8)275-37-28-103(293)256-210(275)308)46-355-415(318,434)396-125-85(377-187(145(125)338-10)277-65-238-105-156(215)226-59-232-162(105)277)48-358-413(316,432)391-120-80(372-182(140(120)333-5)270-32-23-98(214)251-205(270)303)43-354-417(320,436)402-131-94(386-193(151(131)344-16)283-71-244-111-168(283)258-198(221)263-173(111)296)57-366-424(327,443)405-132-91(383-194(152(132)345-17)284-72-245-112-169(284)259-199(222)264-174(112)297)54-363-414(317,433)395-124-84(376-186(144(124)337-9)276-38-29-104(294)257-211(276)309)47-356-416(319,435)397-126-89(381-188(146(126)339-11)278-66-239-106-157(216)227-60-233-163(106)278)52-361-422(325,441)403-133-92(384-195(153(133)346-18)285-73-246-113-170(285)260-200(223)265-175(113)298)55-364-420(323,439)400-130-88(380-192(150(130)343-15)282-70-243-110-161(220)231-64-237-167(110)282)51-360-419(322,438)399-128-90(382-190(148(128)341-13)280-68-241-108-159(218)229-62-235-165(108)280)53-362-423(326,442)404-134-95(387-196(154(134)347-19)286-74-247-114-171(286)261-201(224)266-176(114)299)58-367-425(328,444)406-135-93(385-197(155(135)348-20)287-75-248-115-172(287)262-202(225)267-177(115)300)56-365-421(324,440)401-129-87(379-191(149(129)342-14)281-69-242-109-160(219)230-63-236-166(109)281)50-359-418(321,437)398-127-86(378-189(147(127)340-12)279-67-240-107-158(217)228-61-234-164(107)279)49-357-412(315,431)390-119-78(370-181(139(119)332-4)269-31-22-97(213)250-204(269)302)41-350-408(311,427)388-117-76(39-288)368-179(137(117)330-2)272-34-25-100(290)253-207(272)305;;;;;;;;;;;;;;;;;;;/h21-38,59-95,116-155,178-197,288,295H,39-58H2,1-20H3,(H,310,426)(H,311,427)(H,312,428)(H,313,429)(H,314,430)(H,315,431)(H,316,432)(H,317,433)(H,318,434)(H,319,435)(H,320,436)(H,321,437)(H,322,438)(H,323,439)(H,324,440)(H,325,441)(H,326,442)(H,327,443)(H,328,444)(H2,212,249,301)(H2,213,250,302)(H2,214,251,303)(H2,215,226,232)(H2,216,227,233)(H2,217,228,234)(H2,218,229,235)(H2,219,230,236)(H2,220,231,237)(H,252,289,304)(H,253,290,305)(H,254,291,306)(H,255,292,307)(H,256,293,308)(H,257,294,309)(H3,221,258,263,296)(H3,222,259,264,297)(H3,223,260,265,298)(H3,224,261,266,299)(H3,225,262,267,300);;;;;;;;;;;;;;;;;;;/q;19*+1/p-19/t76-,77-,78-,79-,80-,81-,82-,83-,84-,85-,86-,87-,88-,89-,90-,91-,92-,93-,94-,95-,116-,117-,118-,119-,120-,121-,122-,123-,124-,125-,126-,127-,128-,129-,130-,131-,132-,133-,134-,135-,136-,137-,138-,139-,140-,141-,142-,143-,144-,145-,146-,147-,148-,149-,150-,151-,152-,153-,154-,155-,178-,179-,180-,181-,182-,183-,184-,185-,186-,187-,188-,189-,190-,191-,192-,193-,194-,195-,196-,197-,407?,408?,409?,410?,411?,412?,413?,414?,415?,416?,417?,418?,419?,420?,421?,422?,423?,424?,425?;;;;;;;;;;;;;;;;;;;/m1.................../s1

InChIKey

FZJHBHYSGPESDI-FWLJYILUSA-A

Smiles

[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].CO[C@@H]1[C@H](O)[C@@H](COP(=O)([S-])O[C@@H]2[C@@H](COP(=O)([S-])O[C@@H]3[C@@H](COP(=O)([S-])O[C@@H]4[C@@H](COP(=O)([S-])O[C@@H]5[C@@H](COP(=O)([S-])O[C@@H]6[C@@H](COP(=O)([S-])O[C@@H]7[C@@H](COP(=O)([S-])O[C@@H]8[C@@H](COP(=O)([S-])O[C@@H]9[C@@H](COP(=O)([S-])O[C@@H]%10[C@@H](COP(=O)([S-])O[C@@H]%11[C@@H](COP(=O)([S-])O[C@@H]%12[C@@H](COP(=O)([S-])O[C@@H]%13[C@@H](COP(=O)([S-])O[C@@H]%14[C@@H](COP(=O)([S-])O[C@@H]%15[C@@H](COP(=O)([S-])O[C@@H]%16[C@@H](COP(=O)([S-])O[C@@H]%17[C@@H](COP(=O)([S-])O[C@@H]%18[C@@H](COP(=O)([S-])O[C@@H]%19[C@@H](COP(=O)([S-])O[C@@H]%20[C@@H](CO)O[C@H]([C@@H]%20OC)N%21C=CC(=O)NC%21=O)O[C@H]([C@@H]%19OC)N%22C=CC(=NC%22=O)N)O[C@H]([C@@H]%18OC)n%23cnc%24c%23ncnc%24N)O[C@H]([C@@H]%17OC)n%25cnc%26c%25ncnc%26N)O[C@H]([C@@H]%16OC)n%27cnc%28C(=O)NC(=Nc%27%28)N)O[C@H]([C@@H]%15OC)n%29cnc%30C(=O)NC(=Nc%29%30)N)O[C@H]([C@@H]%14OC)n%31cnc%32c%31ncnc%32N)O[C@H]([C@@H]%13OC)n%33cnc%34c%33ncnc%34N)O[C@H]([C@@H]%12OC)n%35cnc%36C(=O)NC(=Nc%35%36)N)O[C@H]([C@@H]%11OC)n%37cnc%38c%37ncnc%38N)O[C@H]([C@@H]%10OC)N%39C=CC(=O)NC%39=O)O[C@H]([C@@H]9OC)n%40cnc%41C(=O)NC(=Nc%40%41)N)O[C@H]([C@@H]8OC)n%42cnc%43C(=O)NC(=Nc%42%43)N)O[C@H]([C@@H]7OC)N%44C=CC(=NC%44=O)N)O[C@H]([C@@H]6OC)n%45cnc%46c%45ncnc%46N)O[C@H]([C@@H]5OC)N%47C=CC(=O)NC%47=O)O[C@H]([C@@H]4OC)N%48C=CC(=O)NC%48=O)O[C@H]([C@@H]3OC)N%49C=CC(=O)NC%49=O)O[C@H]([C@@H]2OC)N%50C=CC(=NC%50=O)N)O[C@H]1N%51C=CC(=O)NC%51=O