Highlights of key demographic and clinical characteristics of SMA members

Highlights of key demographic and clinical characteristics of SMA members. Interesting new study from Cure SMA – ”An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members. Results : A total of 1,966 individuals with SMA were included in the analysis. Of these… Att fortsätt läsa

What happens to AveXis after the sale

What happens to AveXis after the sale. The sale of AveXis unsettled some SMA patients, but it has now become known. Novartis plans a smooth transition of AveXis operations and the integration of AveXis’ talented and dedicated employees into the Novartis organization to continue the mission of bringing AVXS-101 to… Att fortsätt läsa

Follow-Up Study of Olesoxime in Patients with Type 2 or Non-Ambulatory Type 3 Spinal Muscular Atrophy

New results of the Long Term Study of Oleos (NCT02628743) olesoxime was generally safe and well tolerated at the dose assessed. Maintenance of motor function observed over 2 years in the Phase 2 study was followed by a substantial decline in MFM D1+D2 (>2 points/year) after drug discontinuation. However, the… Att fortsätt läsa

Novartis kjøper AveXis-selskap

Novartis kjøper AveXis-selskap. Läkemedelsbolag Novartis från Basel gör en av sina största miljarder kjöp. Vid mitten av året kommer det att förvärva 8,7 miljarder dollar av genterapi från klinisk stadium från det amerikanska företaget AveXis. Företaget stärker nu sitt monopol inom genterapi för sällsynta sjukdomar och utvecklar på flera fronter… Att fortsätt läsa