DM1

·MeSH: Myotonia

14 drugs in development·7 companies·5 clinical trials

Drug Pipeline by Phase

Phase 3(3)

Drug	Company
DYNE-251	Dyne Therapeutics, Inc. (DYN)
srp-1003	Sarepta Therapeutics, Inc. (SRPT)
zeleciment basivarsen	Dyne Therapeutics, Inc. (DYN)

Phase 2(1)

Drug	Company
WAKIX (pitolisant) - Myotonic Dystrophy (DM1)	Harmony Biosciences Holdings, Inc. (HRMY)

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
aoc 1001	Avidity Biosciences, Inc. (RNAM)
DYNE-101	Dyne Therapeutics, Inc. (DYN)
vx-670	Entrada Therapeutics, Inc. (TRDA)
vx-670	VERTEX PHARMACEUTICALS INC / MA (VRTX)

BioSniper tracks 14 drugs in development for DM1 across 7 companies.

There are 5 clinical trials targeting DM1 tracked by BioSniper.

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