An FDA complete response letter for idebenone in LHON indicates unresolved NDA deficiencies and necessitates additional data before approval can be reconsidered. Priority review status and a February ...

The first group of patients in the GS010 AAC program were treated at the 15-20 National Hospital in Paris on March 19. The REVISE dose-ranging study is proceeding according to the planned ...

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The biotech could run out of funding before it can launch a single product.

RNA splicing involves the removal of non-coding introns and the joining of coding exons in precursor mRNA (pre-mRNA) to produce mature messenger RNA (mRNA). Splicing changes can lead to diseases, ...