Data from DELIVER and ACHIEVE Clinicals Trial to be Presented -- Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on ...

One mutation is an adenine to thymine transversion at position +3 of the intron 6 splice-donor site. Using LMSF mRNA, RT-PCR of a portion of the AR androgen-binding domain yielded a small amount ...

Announcing a new article publication for Cardiovascular Innovations and Applications journal. Alternative splicing (AS), a critical process for gene expression regulation, allows a single precursor ...

Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy: Basel Friday, February 14, 2025, 13:00 Hrs [IST] Roche, announced that the US Food ...

1129–5923C>G splice mutation was significantly enriched in patients with severe 5-FU-related toxicity (9.1%; 66 patients) compared with patients without toxicity (2.2%; 137 patients), confirming ...

Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries ; Evrysdi tablet can be stored at room temperature and offers the same demon ...

Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and sa ...

Sionna Therapeutics has closed its initial public offering, raising $191 million for its pipeline of therapies for cystic ...