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DUX4

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Representative image: Clinicians review muscle imaging and RNA research data as Dyne Therapeutics’ $DYN DYNE-302 receives FDA clearance to enter its first human trial in facioscapulohumeral muscular dystrophy.
Pharma & Biotech

FDA clears DYNE-302 Phase 1 trial targeting DUX4 in facioscapulohumeral muscular dystrophy

FDA clears Dyne’s Phase 1 DYNE-302 trial in FSHD, testing safety, DUX4 suppression and muscle-targeted siRNA delivery.

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Representative image: A clinician reviews muscle MRI scans as Epicrispr Biotechnologies’ EPI-321 delivers early evidence of increased lean muscle volume in patients with facioscapulohumeral muscular dystrophy.
Pharma & Biotech

Epicrispr’s one-time EPI-321 therapy shows early muscle growth after DUX4 silencing

Read how Epicrispr’s early EPI-321 muscle gains could reshape FSHD drug development, and what the small trial still cannot prove.

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