Amyotrophic lateral sclerosis. Rett syndrome. Duchenne muscular dystrophy. Down syndrome. Rare diseases, yes. But devastating nonetheless. Yet, drug companies typically don’t invest in finding treatments for them.
With over 7,000 recognized rare diseases, an estimated 350 million people worldwide are affected at any given time. But, because of the relatively small population afflicted by any one of these illnesses, affected people are often neglected in the search for treatments and cures.
Hope looks like this: A mouse model of spinal muscular atrophy (SMA) developed at JAX was essential to the development of Spinraza, the first drug approved by the FDA to treat newborns, children and adults with SMA.
Yes, we can create better futures for people affected by rare diseases. By creating and distributing mouse models of various genetic diseases to researchers around the world, JAX is making possible widespread testing of potential drug treatments in the same genetic models found in patients. With more scientists armed with better models of rare diseases, treatments and cares are within reach.
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JAX develops and distributes hundreds of mouse models for rare genetic disorders such as cystic fibrosis, Down syndrome, spinal muscular atrophy and ALS. Learn more