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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

ARTHEx Biotech reports ATX-01 brain activity in preclinical myotonic dystrophy study

by Pallavi Madhiraju
July 27, 2026
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ARTHEx Biotech reports peer-reviewed preclinical evidence that ATX-01 reaches the brain and improves DM1-related behavior in mice.

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Latest news and features

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POPULAR NEWS

Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

Niagen wins twin rare-disease designations, but can NB4168 turn nicotinamide riboside evidence into a medicine?

July 14, 2026

Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

Why Mirum Pharmaceuticals and Incyte’s zilurgisertib data could reset expectations in FOP treatment

June 15, 2026

Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

Gilead and Merck pipeline update reveals promise in HIV and pressure in oncology

June 9, 2026

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TAGS

clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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