$SGMO
A new guide from the
#Fabry International Network is aimed at young adults with Fabry disease and clearly illustrates how debilitating the condition can be, far beyond its purely medical symptoms. Those affected often have to live with chronic pain, fatigue, gastrointestinal problems, heart and kidney issues, as well as uncertainty about the long-term course of the disease, from a young age. Added to this are challenges in the transition from paediatric to adult healthcare, as well as in education, career choices, relationships and family planning. The guide addresses precisely these issues and aims to help young people shape their daily lives independently and connect with others affected by the condition.
The publication makes it clear that Fabry is not merely a rare metabolic disorder, but one that has a lasting impact on many areas of life. Against this backdrop, it is understandable why there is such great hope for new therapeutic approaches. A successful one-off gene therapy such as Sangamo’s ST-920 could fundamentally change the lives of many patients: instead of lifelong regular treatments, a long-term or even permanent restoration of the missing enzyme activity could be achieved. This would offer the prospect of significantly reducing the burden of the disease, organ complications and the need for treatment, and enable those affected to lead a much freer and more predictable life.
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