Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the
Ipsen's new chief executive David Loew pledged to ramp up spending on the company's pipeline when he took the helm last year, and a flurry of licensing deals in the last few weeks shows tha
The UK-based AI technology company Healx has begun a partnership with the Foundation for Angelman Syndrome Therapeutics (FAST), focusing on developing new therapies for the rare neurogeneti
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric
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