With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. | Late Thursday, PTC revealed that ...
In a statement, PTC's chief executive, Matthew Klein, said the company had decided to pull the filing for Translarna (ataluren) as a treatment for nonsense mutation DMD after the FDA said it was ...
ATRX, a gene involved in organising and maintaining DNA structure within cells, is commonly altered in neuroblastoma. While the presence of ATRX mutations has long been associated with certain disease ...
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