Nyfødtscreening er så viktig, det kan redde liv, hvis du behandler symptomene tidlig. Å starte en rask behandling er spesielt viktig med autoimmune sykdommer eller genetiske sykdommer som spinal muskelatrofi.
En ny inlegg fra Oslo universitetsykehus
Etikett: Evrysdi (risdiplam)
New clinical trial to Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Roche presents its new data on the SUNFISH, JEWELFISH, RAINBOWFISH study
- Exploratory efficacy data showed 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at two years
- 59% of infants were able to sit without support for at least 5 seconds
- No new safety signals were identified
- In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months and older
- Read more