a good fit for the rare disease drugs from GSK. Aside from Strimvelis, Orchard gets two late-stage clinical programmes in ongoing registrational studies for metachromatic leukodystrophy (MLD ...
GlaxoSmithKline’s gene therapy Strimvelis for the treatment of the ultra-rare ‘bubble baby syndrome’ has been approved by NICE. The one-off therapy costs €594,000, equivalent to £505,000 ...