Rare & Genetic Diseases

A woman holds a newborn baby at the hospital.
| 6 min read
Enzyme replacement therapy has been the mainstay of Pompe disease. New and emerging therapies may provide more options for patients.
Artwork of a piece of DNA with stiches along it.
| 3 min read
New dual AAV vector delivery overcomes challenges in delivering large genes.
A visual representation of an adeno-associated virus used in gene therapies.
| 3 min read
The gene therapy treats the rare genetic disease AADC deficiency by delivering a functional gene into the brain.
A 3D rendering of motor neurons lit up with blue, purple, orange, and green coloring showing synapses against a black background.
An illustration shows epithelial cells in green within a lobule of normal breast tissue, with aneuploid cells highlighted in red.
A doctor holds a young boy’s hands in a doctor’s office.
| 3 min read
A conceptual illustration of a drug capsule filled with microchips, representing the integration of artificial intelligence in drug discovery and development
A 3D illustration of two DNA strands in a transparent bubble
A toddler holds an adult’s hands as she walks outside during an autumn afternoon.
| 9 min read
An illustration showing a DNA strand and scientists removing segments with tweezers, representing the CRISPR gene editing technology
A woman with ichthyosis sits at a table working on a laptop.
| 9 min read
A human ear against a backdrop of blonde hair
| 4 min read
A 3D illustration of protein molecules floating in a dark blue background
| 7 min read
Interconnected neurons send signals to each other.
| 3 min read
A digital rendering of a DNA base being removed from a DNA helix, representing genome editing
Zoomed in 3D-rendering of a DNA helix with overlapping lines in dramatic lighting.
| 4 min read
Clear blue test tubes with DNA inside
| 5 min read
Pink hands cutting and tweezing DNA on a pink background
| 5 min read
A blue iris of an eye is shown against a black background.
| 10 min read
A 3D illustration of DNA
| 5 min read
A compass on a nautical map
Two 96-well plates with the ENFINIA™ DNA logo
| 1 min read
A blue and white brain organoid set against a black background.
| 3 min read
Page 1 of 19 - 369 Total Items
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A 3D rendering of motor neurons lit up with blue, purple, orange, and green coloring showing synapses against a black background.

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A 3D illustration of two DNA strands in a transparent bubble

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Drug Discovery News November 2024 Issue
Latest IssueVolume 20 • Issue 6 • November 2024

November 2024

November 2024 Issue

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