Jax Labs eBook - 21

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The Versatile Mouse Model
for Rare Disease Research
There are more than 7,000 rare (also known as orphan) diseases affecting over
350 million people worldwide. Developing new therapies for rare diseases is very
challenging, given the small patient populations available for clinical trials. Therefore,
animal models, in particular genetically engineered mice, have become one of the most
valuable assets in all of the phases of preclinical orphan drug development.

M

ore than 7,000 rare (or orphan) diseases have been described so far and affect
over 350 million people worldwide. Unfortunately, less than 10% of these
diseases have an approved treatment, and developing new therapies has become
a widely recognized priority by many national authorities.
The development of a new therapy for a rare disease poses some unique challenges,
such as the difficulty to run clinical trials on very small patient populations. As
a consequence of these clinical limitations, animal models, and-specifically-
genetically engineered mice, have become one of the most valuable assets in all of the
phases of preclinical orphan drug development-from gene discovery and validation,
to safety and efficacy studies-and are playing an increasingly important role in driving
clinical experimentation decisions for these class of therapies.

21 | GENengnews.com

A D D I T I O N A L CO N T E N T

Unravel the drug discovery
path with JAX G O THER E



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Jax Labs eBook

Table of Contents for the Digital Edition of Jax Labs eBook

Contents
Jax Labs eBook - 1
Jax Labs eBook - 2
Jax Labs eBook - 3
Jax Labs eBook - Contents
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Jax Labs eBook - 21
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