Scotland opens door to Translarna for Duchenne muscular dystrophyA disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland Share XScotland opens door to Translarna for Duchenne muscular dystrophyhttps://pharmaphorum.com/news/scotland-opens-door-to-translarna-for-duchenne-muscular-dystrophy/
NICE backs Sobi’s Kineret for rare inflammatory diseaseThe UK’s cost-effectiveness agency NICE has recommended that Sobi’s Kineret can be used to treat NHS patients with Share XNICE backs Sobi’s Kineret for rare inflammatory diseasehttps://pharmaphorum.com/news/nice-backs-sobis-kineret-for-rare-inflammatory-disease/
Solving the “information challenge” of rare disease diagnosisGetting a diagnosis is often the biggest challenge facing a rare disease patient in the UK. We take Share XSolving the “information challenge” of rare disease diagnosishttps://deep-dive.pharmaphorum.com/magazine/research-and-development-2021/solving-the-information-challenge-of-rare-disease-diagnosis/
Protein misfolding specialist Gain nets $40m from IPOGain Therapeutics has priced its initial public offering, raising more than $40 million for its computational drug discovery Share XProtein misfolding specialist Gain nets $40m from IPOhttps://pharmaphorum.com/news/protein-misfolding-specialist-gain-nets-40m-from-ipo/
Expediting innovation in the rare disease domainRare diseases affect a fraction of the population – 450 million+ people globally. However, its effects can be Share XExpediting innovation in the rare disease domainhttps://pharmaphorum.com/r-d/views-analysis-r-d/expediting-innovations-in-rare-disease-domain/
Switch data back Chiesi/Protalix long-acting Fabry drugChiesi and Protalix have built the case for their long-acting enzyme replacement therapy (ERT) for Fabry disease PRX-102, Share XSwitch data back Chiesi/Protalix long-acting Fabry drughttps://pharmaphorum.com/news/switch-data-back-chiesi-protalix-long-acting-fabry-drug/
Sobi gets EU nod for Doptelet in rare blood disorder ITPSweden’s Sobi has secured EU approval for Doptelet in primary chronic immune thrombocytopenia (ITP), an indication that the Share XSobi gets EU nod for Doptelet in rare blood disorder ITPhttps://pharmaphorum.com/news/sobi-gets-eu-nod-for-doptelet-in-rare-blood-disorder-itp/
Unlocking the potential in rare disease research with decentralised trialsA survey by rare disease patient network Raremark found that 86% of the community members asked were interested Share XUnlocking the potential in rare disease research with decentralised trialshttps://pharmaphorum.com/views-analysis-patients/unlocking-the-potential-in-rare-disease-research-with-decentralised-trials/
J&J’s Darzalex Faspro is first US therapy for rare blood disorderJohnson & Johnson’s Darzalex Faspro has become the first product approved by the FDA to treat light chain Share XJ&J’s Darzalex Faspro is first US therapy for rare blood disorderhttps://pharmaphorum.com/news/jjs-darzalex-faspro-is-first-us-therapy-for-rare-blood-disorder/
