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/
Sarepta rebounds on data for limb-girdle muscular dystrophy gene therapyShares in Sarepta spiked on positive data for its gene therapy for a form of muscular dystrophy, after Share XSarepta rebounds on data for limb-girdle muscular dystrophy gene therapyhttps://pharmaphorum.com/news/sarepta-rebounds-on-data-for-limb-girdle-muscular-dystrophy-gene-therapy/
NS Pharma steals a march on Sarepta with DMD drugJapan’s Nippon Shinyaku Pharma has filed for approval of its Duchenne muscular dystrophy (DMD) in the US, increasing Share XNS Pharma steals a march on Sarepta with DMD drughttps://pharmaphorum.com/news/ns-pharma-steals-a-march-on-sarepta-with-dmd-drug/
Novartis points to Zolgensma long-term benefits amid data scandalNovartis has new data in hand for spinal muscular atrophy (SMA) gene therapy Zolgensma that it hopes will Share XNovartis points to Zolgensma long-term benefits amid data scandalhttps://pharmaphorum.com/news/novartis-points-to-zolgensmas-long-term-benefits-amid-data-scandal/
Roche preps filings for satralizumab in rare disease NMOSDRoche is two for two in its phase 3 trials programme for satralizumab in the rare disease neuromyelitis Share XRoche preps filings for satralizumab in rare disease NMOSDhttps://pharmaphorum.com/news/roche-preps-filings-for-satralizumab-in-rare-disease-nmosd/
Who will be the biggest pharma players in orphan drugs?New analysis from GlobalData has identified the predicted top 15 pharma players in 2025 for organ drugs, with Share XWho will be the biggest pharma players in orphan drugs?https://pharmaphorum.com/views-analysis-sales-marketing/who-will-be-the-biggest-pharma-players-in-orphan-drugs/
