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Inside Precision Medicine Innovative Vector Helps Gene Therapy Target Rare Muscular Dystrophy

Duchenne muscular dystrophy

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

Innovative Vector Helps Gene Therapy Target Rare Muscular Dystrophy

Anjali A. Sarkar, PhD -
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Man with Duchenne muscular dystrophy sitting in a motorized wheelchair using power controller with degenerated hands
News & Features

Patient Death Results in FDA Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial

Alex Philippidis -
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Illuminating Hope for Rare Disease
News & Features

Illuminating Hope for Rare Disease

[email protected] -
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Smiling female doctor cheer small boy patient
News & Features

CureDuchenne Link: Building a Next-Generation Data-Sharing Platform to Accelerate Drug Development

Kathy Vuksanaj -
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DNA and Brain
News & Features

Immusoft Scores Deal Worth More than $900M With Takeda to Develop CNS Cell Therapies

Helen Albert -
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Motor neuron connecting to muscle fiber to illustrate myasthenia gravis
News & Features

Sarepta Launches Gene Therapy Trial for Duchenne Muscular Dystrophy

Malorye Branca -
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Adeno-associated virus, illustration
News & Features

Adapted Gene Therapy Method Better for Targeting Muscle Diseases

Helen Albert -
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Smiling female doctor cheer small boy patient
Informatics

CureDuchenne Builds Data-Sharing Platform to Accelerate Drug Development

API User -
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Neurons sending pulsating electric impulses and communicating with each other. Concept For Neurological Diseases, Tumors And Brain Surgery
News & Features

Next Steps for Huntington’s Therapeutics Field After Roche, Wave Failures

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

Duchenne Muscular Dystrophy Progression Delayed in Mouse Model with Metabolite Compound

Christina Jackson -
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