Muscular dystrophies

Dual-Vector Editing Maintains Year-Long Function...
CMN Clinical

Jul. 29, 2026

Three boys with Duchenne muscular dystrophy retained broadly stable motor function one year after receiving GEN6050X, an experimental base-editing and...

Epicrispr's EPI-321 Reaches Full Enrolment for...
CMN Clinical

Jul. 13, 2026

Epicrispr Biotechnologies has completed enrolment and dose escalation in the first-in-human trial of EPI-321, an epigenetic CRISPR-based therapy for...

FDA Clears ARCUS Editor for Duchenne Hot-spot
CMN Clinical

Feb. 18, 2026

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...
CMN Briefs

Jun. 9, 2025

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
IndicatorIndicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Limb Girdle Muscular Dystrophy, LGMD, (NCT05588401)
Sponsors:
Simone Spuler, MD
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
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