UK biotech secures FDA designation for rare disease drug
A UK biotech firm founded to develop the world’s first treatment for a rare, incurable and deadly disease has secured its first FDA designation. SynaptixBio was granted a Rare Paediatric Disease (RPD) Designation by the US drug regulator for its TUBB4a leukodystrophy drug. The genetic and debilitating condition which mainly affects babies and young children. […]