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rare disease

This page shows the latest rare disease news and features for those working in and with pharma, biotech and healthcare.

Epizyme wins FDA approval for epithelioid sarcoma treatment

Epizyme wins FDA approval for epithelioid sarcoma treatment

advanced forms of the rare soft tissue disease and are ineligible for a complete resection. ... Epizyme enrolled 62 patients in the cohort and gave them 800 mg of Tazverik twice a day until their disease progressed or they suffered unacceptable toxicity.

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  • Can marketing change the world? Can marketing change the world?

    print; the nuances of chronic conditions, rare disease and major illness. ... The challenge is to maximise it to disrupt health and move the needle of healthcare access, disease outcomes and global mortality.

  • A snapshot of Healx A snapshot of Healx

    A brief overview of the company. Healx is an AI-powered and patient-inspired technology company focused on accelerating the discovery and development of rare disease treatments. ... to grow our multidisciplinary team of in-house rare disease, drug

  • The long and winding road to asthma breakthroughs The long and winding road to asthma breakthroughs

    Nucala, which has the market boost of being licensed for home administration via self- administered auto-injector and prefilled syringe, is running smoothly with approval for the rare disease EGPA in ... One of the strengths of GSK is that we are keen to

  • Integrating patient reported outcomes into clinical trials Integrating patient reported outcomes into clinical trials

    You have to start with an understanding of the impact of the disease on. ... When Blueprint set out to develop avapritinib in systemic mastocytosis there was not a PRO measure for the rare disease, which causes a diverse range of symptoms such as itching,

  • Rare diseases: redefining medical communications Rare diseases: redefining medical communications

    Disease awareness. Given their nature, the index of suspicion for a given rare disease is often very low. ... In addition, the number of experts in any rare disease is typically limited.

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  • emotive supports Rare Disease Day 2019

    With a series of events and activities over the week leading up to Rare Disease Day on February 28th, we showed our support for stimulating greater awareness of Rare Diseases along ... In line with the Rare Disease Day theme, we took photos of our team

  • Porphyria Awareness Week – Round Up

    Porphyria Awareness Week has given the team at emotive an opportunity to show our support for those touched by this debilitating Rare Disease as well as our enthusiasm for working in ... Porphyria is a rare condition that is thought to affect around 1 in

  • Learning about Rare Genetic Disorders on Undiagnosed Children’s Day

    Children can be left facing an uncertain future and an unclear treatment pathway when they have a rare disorder. ... A   Rare Disease UK  report published in 2016 found that nearly half of the respondents who identified as undiagnosed had been waiting

  • After a busy first quarter, emotive welcomes five new team members

    With two exciting new EMEA and Global accounts added in Rare Disease and Advanced Therapies along with expanding existing client work, emotive is delighted to welcome five new recruits to the ... treatment of rare diseases.” said Dorothy at the end of

  • Three amazing women at emotive talk about what they love about medical communications

    Everyone is passionate about their work. Working in rare diseases, where there are so many exciting new advances, I feel good about what we do. ... emotive is an award-winning healthcare communications agency working with leading global life sciences

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