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X-linked genetic diseases

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

Sarepta’s Gene Therapy for DMD Misses on Key Endpoint in Pivotal Trial

Malorye Branca -
0
Colorful rare disease awareness ribbon with a group of different colored sillouettes symbolizing people with rare diseases.
Molecular Dx

Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient

Chris Anderson -
0
Genetic research at the laboratory
Molecular Dx

FDA Approves Hemophilia A Gene Therapy

Malorye Branca -
0
DNA Abstract Background
News & Features

Out of the Gate: Kate Tx Nets $51M And Astellas Gene Therapy Deal

Malorye Branca -
0
DNA structure
News & Features

Five Clinical-Stage Cell and Gene Therapy Startups Leading the Pack

Jonathan Smith -
0
Motor neuron connecting to muscle fiber to illustrate myasthenia gravis
News & Features

Sarepta Gets Adcomm Backing for Duchenne Gene Therapy

Malorye Branca -
0
Motor neuron connecting to muscle fiber to illustrate myasthenia gravis
News & Features

Tevard Inks Collaboration with Vertex on tRNA for Muscular Dystrophy

Malorye Branca -
0
Man with Duchenne muscular dystrophy sitting in a motorized wheelchair using power controller with degenerated hands
Molecular Dx

Duchenne Muscular Dystrophy Gene Linked to Cancer

Helen Albert -
0
Futuristic flight through a digital line landscape, Blue dust particle
Informatics

Real-World Data: The Secret Sauce for Rare Disease Research?

Malorye Branca -
0
Genetic research at the laboratory
Molecular Dx

REGENXBIO Launches Early Phase Trial of Its Gene Therapy for Duchenne Muscular Dystrophy

Malorye Branca -
0
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