Delivering change through better rare disease communicationsOPEN Health’s Richard Jones, Gemma Allen and Charlotte Richards discuss how pharma can utilise tools, channels and stakeholders Share XDelivering change through better rare disease communicationshttps://deep-dive.pharmaphorum.com/magazine/research-and-development-disruption-2020/delivering-change-through-better-rare-disease-communications/
Takeda signs $882m rare disease alliance with Oxford biotech EvoxEvox Therapeutics is the second UK biotech to sign a big deal with a pharma giant this week, Share XTakeda signs $882m rare disease alliance with Oxford biotech Evoxhttps://pharmaphorum.com/news/takeda-signs-882m-rare-disease-alliance-with-oxford-biotech-evox/
Bringing social media listening into R&DAfter her daughter was diagnosed with Prader-Willi syndrome, Maria Picone used social media listening and AI analytics to Share XBringing social media listening into R&Dhttps://deep-dive.pharmaphorum.com/magazine/research-and-development-disruption-2020/bringing-social-media-listening-rd/
The life-changing burden of my rare diseaseI was first diagnosed with a rare autoimmune disease, Sjogren’s Syndrome, about ten years ago, after several years Share XThe life-changing burden of my rare diseasehttps://pharmaphorum.com/views-analysis-patients/the-life-changing-burden-of-my-rare-disease-sarcoidosis/
Girl loses High Court battle over NICE review of BioMarin’s PKU drugAn 11-year-old girl with the rare disease phenylketonuria (PKU) has lost a legal battle in the High Court Share XGirl loses High Court battle over NICE review of BioMarin’s PKU drughttps://pharmaphorum.com/news/girl-loses-high-court-battle-over-nice-review-of-biomarins-pku-drug/
Pfizer’s EU Vyndaqel okay ‘sets up $1.5bn sales next year’Pfizer’s Vyndaqel has become the first drug to be approved for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in Europe, Share XPfizer’s EU Vyndaqel okay ‘sets up $1.5bn sales next year’https://pharmaphorum.com/news/pfizers-eu-vyndaqel-okay-sets-up-1-5bn-sales-next-year/
First UK patients get Novartis’ Luxturna gene therapy for blindnessThe first NHS patients have started treatment with Novartis’ Luxturna, a gene therapy for a sight-robbing inherited disease, Share XFirst UK patients get Novartis’ Luxturna gene therapy for blindnesshttps://pharmaphorum.com/news/first-uk-patients-get-novartis-luxturna-gene-therapy-for-blindness/
Alnylam gets CHMP backing for second gene-silencing drug GivlaariAlnylam could be weeks away from getting its second RNA interference drug approved in Europe, after a CHMP Share XAlnylam gets CHMP backing for second gene-silencing drug Givlaarihttps://pharmaphorum.com/news/alnylam-gets-chmp-backing-for-second-gene-silencing-drug-givlaari/
Ipsen craters as pricey rare disease drug is put on hold againFrench drugmaker Ipsen’s big-ticket investment in rare bone disease drug palovarotene is looking increasingly like a poor deal, Share XIpsen craters as pricey rare disease drug is put on hold againhttps://pharmaphorum.com/news/ipsen-craters-as-pricey-rare-disease-drug-is-put-on-hold-again/
