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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.

Novartis and NHS England reach ‘landmark’ deal for Zolgensma gene therapy for SMA

Novartis and NHS England reach ‘landmark’ deal for Zolgensma gene therapy for SMA

SMA is a rare genetic disease that causes paralysis, muscle weakness and progressive loss of movement. ... As is the case with many new treatments for very rare diseases, limited evidence means there are uncertainties about the long-term benefits of

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Latest appointments

  • Azafaros expands executive team with three senior hires Azafaros expands executive team with three senior hires

    While at Sanofi, she served as head of rare diseases, Central and Eastern Europe – in this role, she led all commercial activities for rare disease-related programmes in those regions.

  • 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

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