We are back from the RNA2107 in Prague. Very exciting talks!
One highlight, Adrian Krainer talked about SPINRAZA, a new therapy against spinal muscular atrophy (SMA) based on antisense oligos. SMA produces the loss of motor neurons that ends up in muscle wasting and early dead (the life expectation of a new born baby with type 1 SMA is ten months). The recently developed treatment treatment has shown excellent results in phase III clinical trials. Adrian guided us through the conception and development of the drug and included emotive videos of how SPINRAZA improves the life of children affected with SMA. An inspiring talk that demonstrates that basic science is fundamental for advancing applied research.