A 3 year-old girl presented with progressive spasticity, visual loss and loss of motor milestones. Metachromatic Leukodystrophy (MLD): Flair axial MRI images. Note the bilateral confluent white matter ...

PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects for gene ...

RNAI start-up City Therapeutics raises $135m, with Purespring, Judo Bio, GEMMABio, and Basecamp Research also completing financings this week.