This article that we wrote with our colleagues from Tuebingen describes the state of the art in ASO treatment options for rare neurological diseases. Both 1 Mutation 1 Medicine (1M1M) and Dutch Center for RNA Therapeutics (DCRT) aim to develop patient-specific ASOs and treat eligible patients within Europe and the Netherlands, respectively. The article offers insight into the challenges and advantages of this type of treatment and describes regulatory issues, patient eligibility criteria, safety studies, ASO development, envisioned treatment and measuring clinical outcomes. It gives a very clear overview of what DCRT and 1M1M have done to prepare for treatment of eligible patients.
posted on January 23, 2023
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