Edaravone may exert its neuroprotective effects in ALS by also correcting the abnormal localization of TDP-43, a key ...

Shares of Sarepta Therapeutics, Inc. (SRPT) traded over 2% lower in afternoon trade on Friday after the company said it has ...

The major workforce overhaul comes a month after parent company Roche deemed the cell and gene therapy firm a financial loss.

Roche’s reorganization of Spark Therapeutics is coming more into focus, with nearly 300 employees being let go by the end of ...

FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

Artis BioSolutions joins a host of startups trying to improve development and manufacturing capacity for cutting-edge gene ...

Pacira BioSciences PCRX announced that it has dosed the first patient in a mid-stage study of pipeline candidate PCRX-201 ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

Shares of Sarepta Therapeutics SRPT dropped more than 6% yesterday after the EMA placed a clinical hold on all studies ...

If you look closely at the fundamental discoveries that have changed human health, physician–scientists have been at the ...

The study, published in The Lancet, a team from the Moorfields Eye Hospital and University College London Institute of ...

Gene therapy and gene editing are on the cutting edge of modern biotechnology. Gene therapies are used to correct genetic abnormalities by introducing genetic material at a cellular level and ...