source
- Atsena reports early efficacy signals for gene therapy ATSN-201 in X-linked retinoschisis trialAtsena Therapeutics presented data from the early parts of its Lighthouse gene therapy trial for X-linked retinoschisis (XLRS) at the American Academy of Ophthalmology meeting. The trial administe…
- Experimental exon-skipping drug from Avidity Biosciences shows early improvement in a Duchenne patientA recent story follows two teenage boys living with Duchenne muscular dystrophy, a rare and fatal muscle‑wasting condition. While one boy’s health continues to decline, the other, a 12‑year‑old nam…