Technology provides hope for rare diseases
As part of this year’s Jeans for Genes day Healx founder, Tim Guilliams, highlights how new technology is offering hope to rare disease patients. With over 3.5 million people in the UK living with a rare disease the need to offer better treatments is paramount.
In the article Tim discusses recent drug repurposing developments as a potential cure for a seven-year-old American boy diagnosed with NGLY-1 deficiency. A disease caused by a mutated gene that leads to developmental delay and reduced muscle mass.
The money raised from this event funds Genetic Disorders UK, the national charity dedicated to supporting families affected by genetic disorders.
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