Muscular dystrophies

FDA Clears ARCUS Editor for Duchenne Hot-spot

The US FDA has issued a Study May Proceed notification for PBGENE-DMD, Precision BioSciences' investigational in vivo gene-editing therapy for...

Modalis Partners with SOLVE FSHD to Develop CRISPR...

Modalis Therapeutics has partnered with SOLVE FSHD to advance the development of an experimental epigenome-editing therapy for facioscapulohumeral...

Muscular dystrophies

Merosin-deficient congenital muscle dystrophy type 1a, MDC1a, (NCT06582537)
Sponsors:
Maastricht University

IND Enabling

Phase I

Phase II

Phase III

Facioscapulohumeral Muscular Dystrophy, (NCT06907875)
Sponsors:
Epicrispr Biotechnologies

IND Enabling

Phase I

Phase II

Phase III

Duchenne Muscular Dystrophy, DMD, (NCT06594094)
Sponsors:
HuidaGene Therapeutics Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Duchenne Muscular Dystrophy, DMD, (NCT05514249)
Sponsors:
Cure Rare Disease, Inc

IND Enabling

Phase I

Phase II

Phase III

Limb Girdle Muscular Dystrophy, LGMD, (NCT05588401)
Sponsors:
Simone Spuler, MD

IND Enabling

Phase I

Phase II

Phase III

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