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rare diseases

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

Launch leaders’ blog part 1

Launch leaders’ blog part 1

a solution to the global coronavirus pandemic, as well as to many other rare and not so rare diseases.

Latest news

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Approximately 121 fully matching, plus 271 partially matching documents found.

Latest Intelligence

  • Biopharma and orphan drugs Biopharma and orphan drugs

    Now there are over 160 orphan medicinal products approved by the EMA for more than 120 rare diseases. ... Also, there are significant clinical differences between trial subjects in many rare diseases.

  • Staying ahead in the era of precision medicine Staying ahead in the era of precision medicine

    It’s revolutionary and it’s evolutionary.”. While it is most advanced in oncology, precision medicine has wider, exciting applications beyond oncology and late-stage disease, such as rheumatology, and rare ... Empowering and uniting patient

  • 20 for 2020 – Five pharma leaders to watch in 2020 20 for 2020 – Five pharma leaders to watch in 2020

    to 2025 built on the Prevnar franchise, rare disease therapy Vyndaqel (tafamidis), breast cancer therapy Ibrance (palbociclib) and arthritis drug Xeljanz (tofacitinib), among others. ... In the meantime, Sanofi is stepping up its focus in other areas

  • Bringing the trial to the patient Bringing the trial to the patient

    In addition, clinical trials for rare diseases can be so geographically dispersed that patients may need to travel very long distances to participate in a trial.

  • Novo Nordisk awakens its ‘Sleeping Beauty’ Novo Nordisk awakens its ‘Sleeping Beauty’

    Helfgott stressed the potential: “It is a sleeping beauty. We now have the tools to turn a healthy business unit into a leading one in the rare endocrine and rare blood ... we are going to make a difference in the very difficult area of rare diseases.

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Latest appointments

  • Porter Novelli adds Amber Tovey to London health team Porter Novelli adds Amber Tovey to London health team

    In her new role at Porter Novelli, Tovey will work across a global pharmaceutical client portfolio that includes neurology, rare diseases and HIV.

  • New hires at Crescendo, Healx and Galecto New hires at Crescendo, Healx and Galecto

    Hall (pictured left) has over 25 years of experience in drug development, with the last decade spent focused on rare diseases. ... He co-founded the rare disease patient group support charity Findacure with Nick Sireau, the advisor to and the inspiration

  • New hires at bluebird bio, BioNTech and Provention Bio New hires at bluebird bio, BioNTech and Provention Bio

    Jason's extensive commercial experience, including the planning, preparation and execution of product launches in rare diseases and nascent markets, will be critical as we work to bring teplizumab to type

  • New hires at FibroGen, Saniona and Turbine New hires at FibroGen, Saniona and Turbine

    Saniona . Saniona, a clinical stage biotech focused on rare diseases related to eating disorders and the central nervous system, has appointed Rami Levin (pictured below, right) as chief executive officer. ... He is a veteran biotech leader, with over

  • New faces at Lucid Group and ZPB Associates New faces at Lucid Group and ZPB Associates

    Most recently, she worked in the consultancy’s rare diseases division, where is provided crisis management support in pricing/access barriers for an orphan drug.

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Latest from PMHub

More from PMHub
Approximately 18 fully matching, plus 71 partially matching documents found.

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