Duchenne

Rare Disease·MeSH: Muscular Dystrophy, Duchenne

Prevalence

6.0 / 100K

Incidence

1.5 / 100K/yr

53 drugs in development·22 companies·2 clinical trials

Drug Pipeline by Phase

Approved(11)

Drug	Company
agamree	CATALYST PHARMACEUTICALS, INC. (CPRX)
AGAMREE®	CATALYST PHARMACEUTICALS, INC. (CPRX)
amondys 45	Sarepta Therapeutics, Inc. (SRPT)
amondys 45	Sarepta Therapeutics, Inc. (SRPT)
deflazacort	Amneal Pharmaceuticals, Inc. (AMRX)
Emflaza	PTC THERAPEUTICS, INC. (PTCT)
exondys 51	Sarepta Therapeutics, Inc. (SRPT)
exondys 51	Sarepta Therapeutics, Inc. (SRPT)
Translarna	PTC THERAPEUTICS, INC. (PTCT)
vyondys 53	Sarepta Therapeutics, Inc. (SRPT)
vyondys 53	Sarepta Therapeutics, Inc. (SRPT)

Phase 3(14)

Drug	Company
aoc 1044	Avidity Biosciences, Inc. (RNAM)
Ataluren	PTC THERAPEUTICS, INC. (PTCT)
casimersen	Sarepta Therapeutics, Inc. (SRPT)
duchenne muscular dystrophy"	CAPRICOR THERAPEUTICS, INC. (CAPR)
edsp	Quince Therapeutics, Inc. (QNCX)
eteplirsen	Sarepta Therapeutics, Inc. (SRPT)
golodirsen	Sarepta Therapeutics, Inc. (SRPT)
RGX-202	REGENXBIO Inc. (RGNX)
srp-4045	Sarepta Therapeutics, Inc. (SRPT)
srp-4053	Sarepta Therapeutics, Inc. (SRPT)
srp-5051	Sarepta Therapeutics, Inc. (SRPT)
zeleciment basivarsen (z-basivarsen)	Dyne Therapeutics, Inc. (DYN)
zeleciment rostudirsen	Dyne Therapeutics, Inc. (DYN)
zeleciment rostudirsen (z-rostudirsen)	Dyne Therapeutics, Inc. (DYN)

Phase 2(10)

Drug	Company
Deramiocel	CAPRICOR THERAPEUTICS, INC. (CAPR)
Dyscorban®	CUMBERLAND PHARMACEUTICALS INC (CPIX)
EDG-5506	Edgewise Therapeutics, Inc. (EWTX)
Ezutromid	Summit Therapeutics Inc. (SMMT)
ifetroban	CUMBERLAND PHARMACEUTICALS INC (CPIX)
iv infusion	PepGen Inc. (PEPG)
pgn-edo51	PepGen Inc. (PEPG)
Vasculan	CUMBERLAND PHARMACEUTICALS INC (CPIX)
vesleteplirsen	Sarepta Therapeutics, Inc. (SRPT)
WVE-N531	Wave Life Sciences Ltd. (WVE)

Phase 1(6)

Drug	Company
delandistrogene moxeparvovec	Sarepta Therapeutics, Inc. (SRPT)
INS1201	INSMED Inc (INSM)
KER-065	Keros Therapeutics, Inc. (KROS)
PLN-101325	PLIANT THERAPEUTICS, INC. (PLRX)
rinvatercept	Keros Therapeutics, Inc. (KROS)
SMT C1100	Summit Therapeutics Inc. (SMMT)

Early Phase 1(1)

Drug	Company
sat-3247	Satellos Bioscience Inc. (MSLE)

Phase 1/Phase 2(9)

Drug	Company
bmn 351	BIOMARIN PHARMACEUTICAL INC (BMRN)
entr-601-44	Entrada Therapeutics, Inc. (TRDA)
entr-601-45	Entrada Therapeutics, Inc. (TRDA)
entr-601-50	Entrada Therapeutics, Inc. (TRDA)
entr-601-51	Entrada Therapeutics, Inc. (TRDA)
entr-801	Entrada Therapeutics, Inc. (TRDA)
pbgene-dmd	PRECISION BIOSCIENCES INC (DTIL)
SGT-001	Solid Biosciences Inc. (SLDB)
SGT-003	Solid Biosciences Inc. (SLDB)

Phase 4(2)

Drug	Company
antibiotics	Sarepta Therapeutics, Inc. (SRPT)
elevidys	Sarepta Therapeutics, Inc. (SRPT)

BioSniper tracks 53 drugs in development for Duchenne across 22 companies.

There are 2 clinical trials targeting Duchenne tracked by BioSniper.

The estimated prevalence of Duchenne is approximately 6.0 per 100,000 population.

Full trial data, competitive analysis, and AI-powered pipeline tracking.