Myotonic Dystrophy Type 1

Rare Disease·MeSH: Myotonic Dystrophy

Prevalence

5.0 / 100K

15 drugs in development·8 companies·2 clinical trials

Drug Pipeline by Phase

Phase 3(4)

Drug	Company
del-desiran	Avidity Biosciences, Inc. (RNAM)
DYNE-251	Dyne Therapeutics, Inc. (DYN)
srp-1003	Sarepta Therapeutics, Inc. (SRPT)
zeleciment basivarsen	Dyne Therapeutics, Inc. (DYN)

Phase 2(2)

Drug	Company
rhIGF-I/rhIGFBP-3	INSMED Inc (INSM)
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(3)

Drug	Company
DYNE-101	Dyne Therapeutics, Inc. (DYN)
vx-670	VERTEX PHARMACEUTICALS INC / MA (VRTX)
vx-670	Entrada Therapeutics, Inc. (TRDA)

BioSniper tracks 15 drugs in development for Myotonic Dystrophy Type 1 across 8 companies.

There are 2 clinical trials targeting Myotonic Dystrophy Type 1 tracked by BioSniper.

The estimated prevalence of Myotonic Dystrophy Type 1 is approximately 5.0 per 100,000 population.

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