Pfizer acquires Therachon and its dwarfism drugPfizer has announced it will pay $340 million upfront to snap up rare disease biotech Therachon, which is Share XPfizer acquires Therachon and its dwarfism drughttps://pharmaphorum.com/news/pfizer-acquires-therachon-and-its-dwarfism-drug/
Fulcrum repurposes failed GSK drug for rare diseaseGSK’s failed cardiovascular drug losmapimod looks like it might get a new lease of life after being picked Share XFulcrum repurposes failed GSK drug for rare diseasehttps://pharmaphorum.com/news/fulcrum-repurposes-failed-gsk-drug-for-rare-disease/
Second death in gene therapy trial prompts investigationA second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – Share XSecond death in gene therapy trial prompts investigationhttps://pharmaphorum.com/news/second-death-in-novartis-gene-therapy-trial-prompts-investigation/
Ipsen launches new syringe for rare disease blockbusterIpsen will be launching a new pre-filled syringe for its blockbuster rare disease drug Somatuline (lanreotide) in the Share XIpsen launches new syringe for rare disease blockbusterhttps://pharmaphorum.com/news/ipsen-launches-new-syringe-for-rare-disease-blockbuster/
Sanders calls on FDA to allow low-cost rivals to $375k Catalyst drugUS Senator Bernie Sanders says the FDA should allow pharmacies and generic manufacturers to make low-cost copies of Share XSanders calls on FDA to allow low-cost rivals to $375k Catalyst drughttps://pharmaphorum.com/news/sanders-calls-on-fda-to-allow-low-cost-rivals-to-375k-catalyst-drug/
Gene addition – a new chapter in treating rare diseasesThe scientific advancements of the past 30 years have transformed our understanding of the potential of gene therapy and Share XGene addition – a new chapter in treating rare diseaseshttps://pharmaphorum.com/views-and-analysis/gene-addition-a-new-chapter-in-treating-rare-diseases/
Increasing patient involvement in research to tackle unmet needIt could be said that there is no greater unmet need than a complete and absolute absence of treatment Share XIncreasing patient involvement in research to tackle unmet needhttps://pharmaphorum.com/views-and-analysis/increasing-patient-involvement-research-tackle-unmet-need/
Dutch biotech Pharming plans to challenge big pharma in rare diseasesThe introduction of Shire’s preventive treatment for hereditary angioedema has shaken up the market for drugs for this Share XDutch biotech Pharming plans to challenge big pharma in rare diseaseshttps://pharmaphorum.com/r-d/views-analysis-r-d/pharming-challenge-pharma-rare-diseases/
Rare Youth Project: Can you empower the next generation of rare disease advocates?The next generation of rare disease advocates need a little support in getting their voices heard as part Share XRare Youth Project: Can you empower the next generation of rare disease advocates?https://pharmaphorum.com/views-analysis-patients/rare_youth_project_empower-next-generation-rare-disease-advocates/
