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Inside Precision Medicine FDA Clears Solid Biosciences Neurodegeneration Gene Therapy IND

Genetic diseases

Nervous and cardiovascular system, illustration
News & Features

FDA Clears Solid Biosciences Neurodegeneration Gene Therapy IND

Jonathan D. Grinstein, PhD
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Human kidney cross section on scientific background
News & Features

Better AAV Delivery for Kidney Gene Therapy

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DNA blood cell on scientific background
News & Features

Beam Therapeutics Sickle Cell Base Editing Works, Not Cause of Patient Death

Jonathan D. Grinstein, PhD
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Sickle Cell disease - 3D Illustration of sickled blood cells on a dark background
News & Features

New CMS Payment Model Launched for Sickle Cell Gene Therapy

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Close-up Of Pregnant African American Woman Having Blood Taken for NIPT
Molecular Dx

Abnormal or Non-Reportable NIPT Can Help Detect Cancer During Pregnancy

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Molecule of mRNA, illustration
Precision Medicine

Innovative Packaging of mRNA Therapeutics Improves Their Delivery and Speeds Development

Anita Chakraverty
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Man with Duchenne muscular dystrophy sitting in a motorized wheelchair using power controller with degenerated hands
News & Features

REGENXBIO Gene Therapy Advances to Pivotal Trials in DMD  

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Malignant peripheral nerve sheath tumors (MPNST)
News & Features

In “Quantum Leap,” Almost Half of Adults and Children with NF1 Respond to MEK Inhibitor

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Man with Duchenne muscular dystrophy sitting in a motorized wheelchair using power controller with degenerated hands
News & Features

Sizing it Up: New Method Enables Large Gene Delivery to Treat Muscular Dystrophies

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Human red blood cells x 1000
News & Features

Universal Gene Therapy for Blood Disorders Targets Downstream Pathways

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