Myotonic Dystrophy

Rare Disease·MeSH: Myotonic Dystrophy

Prevalence

5.0 / 100K

24 drugs in development·11 companies·6 clinical trials

Drug Pipeline by Phase

Phase 3(5)

Drug	Company
del-desiran	Avidity Biosciences, Inc. (RNAM)
DYNE-251	Dyne Therapeutics, Inc. (DYN)
rhIGF-I/rhIGFBP-3	INSMED Inc (INSM)
srp-1003	Sarepta Therapeutics, Inc. (SRPT)
zeleciment basivarsen	Dyne Therapeutics, Inc. (DYN)

Phase 2(3)

Drug	Company
pgn-edodm1	PepGen Inc. (PEPG)
pgn-edodm1 for infusion	PepGen Inc. (PEPG)
vx-670	VERTEX PHARMACEUTICALS INC / MA (VRTX)

Phase 1(4)

Drug	Company
ARO-DM1	ARROWHEAD PHARMACEUTICALS, INC. (ARWR)
aro-dm1 intravenous infusion	ARROWHEAD PHARMACEUTICALS, INC. (ARWR)
aro-dm1 subcutaneous	ARROWHEAD PHARMACEUTICALS, INC. (ARWR)
Peripheral Blood Stem Cell Infusion	ARROWHEAD PHARMACEUTICALS, INC. (ARWR)

Preclinical(6)

Drug	Company
aro-dm1	Sarepta Therapeutics, Inc. (SRPT)
DYNE-244	Dyne Therapeutics, Inc. (DYN)
DYNE-245	Dyne Therapeutics, Inc. (DYN)
DYNE-253	Dyne Therapeutics, Inc. (DYN)
DYNE-255	Dyne Therapeutics, Inc. (DYN)
patrol platform	NeuBase Therapeutics, Inc. (NBSE)

Phase 1/Phase 2(4)

Drug	Company
DYNE-101	Dyne Therapeutics, Inc. (DYN)
sar446268	Sanofi (SNYNF)
sar446268	Sanofi (SNY)
vx-670	Entrada Therapeutics, Inc. (TRDA)

Phase 4(2)

Drug	Company
Lidocaine Infusion	ARROWHEAD PHARMACEUTICALS, INC. (ARWR)
Nefopam 120mg/day infusion	ARROWHEAD PHARMACEUTICALS, INC. (ARWR)

BioSniper tracks 24 drugs in development for Myotonic Dystrophy across 11 companies.

There are 6 clinical trials targeting Myotonic Dystrophy tracked by BioSniper.

The estimated prevalence of Myotonic Dystrophy is approximately 5.0 per 100,000 population.

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