Big names in the pharmaceutical industry come together to fight rare diseases
Angiochem and GlaxoSmithKline will soon be working together to develop new treatments for patients of rare diseases with the goal of restoring enzyme function in the brain. Biotechs are looking for new strategies to combat certain diseases involving lysosomal storage because current therapies only do so much and can’t stop a patient’s decline.
GlaxoSmithKline and Angiochem hope to find a way to use the LRP-1 mediated pathway to get drugs from a patient’s blood to their brain tissue. This is a huge breakthrough that would help reverse negative effects on the brain, namely decreased or halted enzyme function.
Some of the genetic disorders that would be helped by this would include Fabry disease, Gaucher’s disease and Pompe disease. Representatives of both companies have shown to be optimistic about the endeavor, and with any luck it won’t be long before this optimism is translated into results.
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