LentiGlobin gene therapy for sickle cell on course for filing, says bluebirdIt has been an amazing year for patients with sickle cell disease (SCD) with two new drug approvals Share XLentiGlobin gene therapy for sickle cell on course for filing, says bluebirdhttps://pharmaphorum.com/news/lentiglobin-gene-therapy-for-sickle-cell-on-course-for-filing-says-bluebird/
Novartis pulls rival to Roche’s risdiplam in SMA, says market limitedWhile Roche’s oral risdiplam has been tipped by some analysts to be a big contender in the market Share XNovartis pulls rival to Roche’s risdiplam in SMA, says market limitedhttps://pharmaphorum.com/news/novartis-pulls-rival-to-roches-risdiplam-in-sma-says-market-limited/
Roche trumpets data for Soliris rival in NMOSD after US, EU filingsRoche has continued to build the case for satralizumab, its experimental therapy for rare disease neuromyelitis optica spectrum Share XRoche trumpets data for Soliris rival in NMOSD after US, EU filingshttps://pharmaphorum.com/news/roche-trumpets-data-for-soliris-rival-in-nmosd-after-us-eu-filings/
Mereo’s brittle bone drug likely to shrug off phase 2b fail, say analystsMereo’s drug candidate for brittle bone disease missed its primary endpoint in a recent phase 2b study, but Share XMereo’s brittle bone drug likely to shrug off phase 2b fail, say analystshttps://pharmaphorum.com/news/mereos-brittle-bone-drug-likely-to-shrug-off-phase-2b-fail-say-analysts/
AstraZeneca, Merck cue up FDA verdict on selumetinib next yearAfter failing to make the grade in multiple cancer types, AstraZeneca and Merck & Co/MSD’s selumetinib could get Share XAstraZeneca, Merck cue up FDA verdict on selumetinib next yearhttps://pharmaphorum.com/news/astrazeneca-merck-cue-up-fda-verdict-on-selumetinib-next-year/
UniQure presses go on Huntington’s gene therapy trialUniQure has started screening patients for its phase 1/2 trial of gene therapy AMT-130 for Huntington’s disease, and Share XUniQure presses go on Huntington’s gene therapy trialhttps://pharmaphorum.com/news/uniqure-presses-go-on-huntingtons-gene-therapy-trial/
Reata rockets on data for Nrf2 drug reacquired from AbbVieReata Pharma has reported positive clinical results with one of its Nrf2 activators for mitochondrial diseases, just days Share XReata rockets on data for Nrf2 drug reacquired from AbbViehttps://pharmaphorum.com/news/reata-rockets-on-data-for-nrf2-drug-reacquired-from-abbvie/
Alexion stalks rare disease drug from Stealth with $30m opt-in dealAlexion has taken an option on a rare disease drug from Stealth Therapeutics in late-stage development, bolstering its Share XAlexion stalks rare disease drug from Stealth with $30m opt-in dealhttps://pharmaphorum.com/news/alexion-stalks-rare-disease-drug-from-stealth-with-30m-opt-in-deal/
Clinuvel gets approval in US for rare skin disorder drug ScenesseAustralian biotech Clinuvel has claimed a US approval for Scenesse, a treatment for a rare skin disorder that Share XClinuvel gets approval in US for rare skin disorder drug Scenessehttps://pharmaphorum.com/news/clinuvel-gets-approval-in-us-for-rare-skin-disorder-drug-scenesse/
