The n-Lorem Foundation and the EspeRare Foundation announced today a strategic collaboration to expand access in Europe to ...

NEW YORK – The European Commission has approved a high-dose regimen of Biogen's Spinraza (nusinersen), an antisense oligonucleotide for 5q spinal muscular atrophy (SMA).

Currently available therapies such as niacin and PCSK9 inhibitors such as Repatha (evolocumab) and Praluent (alirocumab) can reduce Lp (a) levels, but their impact is modest. Another challenge in ...

PacBio (NASDAQ: PACB), developer of the world's most advanced sequencing technologies, today announced plans to pursue a strategic collaboration with the n-Lorem Foundation and EspeRare that we ...

An alternative splicing regulator previously implicated in calcium signaling in the heart ensures cell-type-specific mRNA processing of long neuronal mRNAs in the mouse brain.

Detailed price information for Wave Life Sci Ord Sh (WVE-Q) from The Globe and Mail including charting and trades.

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing ...

Aperture Therapeutics is advancing APRTX-003, an RNA-based antisense therapy targeting MMP9, as a potential ALS treatment.

Arcturus Therapeutics Holdings Inc. (the "Company", "Arcturus", Nasdaq: ARCT), a commercial messenger RNA medicines company ...

Pancreatic ductal adenocarcinoma (PDAC) is the most lethal form of pancreas cancer. It's also the most common form of the ...

Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; ...