56 assets in Duchenne Muscular Dystrophy

Viltolarsen

Aoc 1044

Delandistrogene Moxeparvovec

Golodirsen

Eteplirsen

Prednisolone

Casimersen

Vamorolone

Tas-205

Tadalafil

Ataluren

Givinostat

Deramiocel

Dyne-251

Sgt-003

All-P-Ambo-3'-Deoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2’A?5’)-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2’A?5’)-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secoguanylyl-(2’A?5’)-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secoguanylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2’A?5’)-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secocytidylyl-(2’A?5’)-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2’A?5’)-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secocytidylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secocytidylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secoguanylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,3'-Dideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secothymidylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secoadenylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secoguanylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secocytidylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secocytidylyl-(2'A?5')-P,2',3'-Trideoxy-P-Dimethylamino-2',3'-Imino-2',3'-Secoadenylyl-(2'A?5')-2',3'-Trideoxy-2',3'-Imino-2',3'-Secocytidine

ds-5141b

Sirolimus

Bmn 351

Sat-3247

Wve-N531

Beatrolol

Zeleciment Rostudirsen

Recombinant, Non-Replicating Adeno-Associated Virus Serotype 8 (Raav8) Vector, Encoding A Codon-Optimised Miniaturised Version Of Human Dystrophin (Microdystrophin Variant Abd1-H1-R1-R2-R3-H3-R24-H4-Cr-Ct194), Under Control Of A Synthetic Muscle-Specific Promoter (Spc5-12) And A Small Synthetic Polyadenylation Signal; Flanked By Aav2 Inverted Terminal Repeats. The Expressed Microdystrophin Variant Contains The Actin-Binding Domain 1 (Abd1), Four Rod Domains (R1-3, R24), Three Hinge Domains (H1, H3, H4) And A Cysteine Rich (Cr) Domain; It Also Features The Proximal Portion Of The C-Terminal Domain, Amino Acids 3361-3554 (Ct194)

Avicursen

en001

Pgn-Edo51

Satralizumab

Sevasemten

PF-06939926 IS A RECOMBINANT AAV VECTOR CONTAINING THE AAV SEROTYPE 9 (AAV9) CAPSID PACKAGING A MINIATURIZED CODON-OPTIMIZED VERSION OF THE DMD GENE(CALLED OPTI-DYS.DELTA.3978) WHICH ENCODES THE DOMAINS MINIMALLY REQUIRED FOR FUNCTIONALITY OF THE DYSTROPHIN PROTEIN (MINI-DYSTROPHIN). A SYNTHETIC POLYADENYLATION (SPOLYA) SEQUENCE FOLLOWS THE OPTI-DYS.DELTA.3978 GENE. CARDIAC AND SKELETAL MUSCLE-SPECIFIC TRANSGENE EXPRESSION IS MEDIATED BY A SYNTHETIC ENHANCER AND PROMOTER. THE EXPRESSION CONSTRUCT IS FLANKED BY THE AAV2 INVERTED TERMINAL REPEATS (ITRS). THE 3978 BASEPAIR MINI-DYSTROPHIN OPEN READING FRAME ENCODES THE N-TERMINUSHINGE REGIONS H1, H3 AND H4SPECTRIN-LIKE REPEATS R1, R2 AND R22-24AND THE C-TERMINAL CYSTEINE-RICH REGION OF THE DYSTROPHIN PROTEIN.

Pbgene-Dmd

Entr-601-44

Entr-601-45

Gnr-097

Ncnp-03

NS-050

Phase 1, Sqy51

Semaglutide

Sgt-001

grt6019

hg302

Crd-Tmh-001

Imlifidase

JWK007

Myopaxon

Povidone

Tacrolimus

Bbm-D101

gen6050x intravenous

le051