A nonsense mutation is a genetic mutation in a DNA sequence that results in a shorter, unfinished protein product. DNA is a chain of many smaller molecules called nucleotides. During protein ...
Many human diseases are caused by point mutations that affect RNA splicing. It has been observed that nonsense mutations that generate premature termination of translation products can drive the use ...
Alltrna’s CEO Michelle Warner said that the approach allows for basket trials that can group patients with different diseases together.
Shares of PTC Therapeutics fell after the company said regulators for the European Union won't renew authorization for its Translarna treatment for nonsense mutation Duchenne muscular dystrophy.
A genetic change that boosts a cell's aerobic capacity while also protecting it from excess stress could explain how horses became such powerful athletes, according to a new study in Science.
PTC Therapeutics (PTCT) announced that the European Commission has adopted the opinion of the Committee for Medicinal Products for Human Use of ...
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines ...