Life with multiple myelomaMultiple myeloma patient Bob Munro (pictured below) explains what it’s like to live with the rare blood cancer and Share XLife with multiple myelomahttps://pharmaphorum.com/partner-content/life-with-multiple-myeloma/
Akcea’s rare disease drug Waylivra way too pricey for NHS, says NICELess than a week into 2020 and NICE has provisionally ruled against NHS funding for another drug – Share XAkcea’s rare disease drug Waylivra way too pricey for NHS, says NICEhttps://pharmaphorum.com/news-market-access/akceas-rare-disease-drug-waylivra-way-too-pricey-for-nhs-says-nice/
Alnylam to file rare kidney disease drug after trial winAfter years where RNA interference research was in the doldrums, Alnylam is on a roll and is preparing Share XAlnylam to file rare kidney disease drug after trial winhttps://pharmaphorum.com/news/alnylam-to-file-rare-kidney-disease-drug-after-phase-3-win/
Could Spark Therapeutics’ merger with Roche finally get the go-ahead?Spark Therapeutics’ merger with Roche has moved a step closer after the UK’s competition watchdog okayed the $4.8 Share XCould Spark Therapeutics’ merger with Roche finally get the go-ahead?https://pharmaphorum.com/news/could-spark-therapeutics-merger-with-roche-finally-get-the-go-ahead/
How does prevalence affect pricing of rare disease drugs?New research from CRA looks at the relationship between rarity of a disease and the price countries are Share XHow does prevalence affect pricing of rare disease drugs?https://pharmaphorum.com/views-analysis-market-access/prevalence-pricing-rare-disease-drugs/
Sanofi axes diabetes R&D, focuses on ‘transformative’ therapiesTake a look at Sanofi’s balance sheet and it’s obvious which way the wind is blowing – sales Share XSanofi axes diabetes R&D, focuses on ‘transformative’ therapieshttps://pharmaphorum.com/news/sanofi-axes-diabetes-and-cardio-rd-shifts-focus-on-transformative-therapies/
4th Annual Gene Therapy for Rare DisordersRealize The Commercial Potential of Gene Therapies Share X4th Annual Gene Therapy for Rare Disordershttps://pharmaphorum.com/events/4th-annual-gene-therapy-for-rare-disorders/
FDA approves Global Blood Therapeutics’ sickle cell pillGlobal Blood Therapeutics has seen its sickle cell disease drug approved by the FDA, promising to be the Share XFDA approves Global Blood Therapeutics’ sickle cell pillhttps://pharmaphorum.com/news/fda-approves-global-blood-therapeutics-sickle-cell-pill/
FCS: A life-limiting conditionGP Karishma Patel tells us about the daily challenges of living with the ultra-rare condition Familial Chylomicronaemia Syndrome Share XFCS: A life-limiting conditionhttps://pharmaphorum.com/views-analysis-patients/fcs-a-life-limiting-condition/
