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FSHD

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ARTHEx Biotech’s ATX-01 preclinical research examines whether systemic RNA therapy can reach the brain and improve DM1-related molecular and behavioral abnormalities. Representative image.
Pharma & Biotech

Sarepta’s RNA therapy reaches the nucleus in myotonic dystrophy models. Can it restore muscle function?

Sarepta has reported up to 80% knockdown of DMPK RNA in both the nucleus and cytoplasm of patient-derived muscle cells, alongside correction of abnormal RNA splicing in preclinical models. The findings address a central delivery challenge in myotonic dystrophy type 1, while an ongoing Phase 1/2 trial will determine whether the mechanism translates into patient

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Representative image of pharmaceutical compounds and clinical tools, symbolizing innovation in rare disease drug development. Atossa’s (Z)-Endoxifen orphan drug designation for Duchenne muscular dystrophy reflects broader biotech interest in repositioning oncology assets for pediatric neuromuscular conditions.
Pharma & Biotech

Altay Therapeutics closes $37m Series A for FSHD treatment DX5057

Altay Therapeutics has closed a $37 million Series A financing to advance DX5057, an investigational oral DUX4 inhibitor for facioscapulohumeral muscular dystrophy. The company expects to begin Phase 1 testing in early 2027, following preclinical evidence of reduced muscle injury and preserved function.

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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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