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features: Cell and Gene Therapy

First ever targeted treatment for SOD1-ALS: FDA approve tofersen

On April 25th, 2023, the U.S. Food and Drug Administration (FDA) approved tofersen (BIIB067) for the treatment of amyotrophic lateral…

AMT-130: the first gene therapy on the horizon for Huntington’s disease?

The first interim data has been published from a Phase I/II trial testing the safety and tolerability of AMT-130. If…

New in epilepsy: promising therapeutic strategies

New treatment strategies are gaining attention in epilepsy, where current therapeutic options are ineffective for many. Learn more about the…