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rare disease unit

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

Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

Two patients had at least one year of follow-up and showed modest increases in the NorthStar Ambulatory Assessment (NSAA) scale, used to assess symptoms caused by the muscle-wasting disease. ... Pfizer adopted a cautious tone in its assessment of the data

Latest news

  • Europe responding to gene therapy challenge, but picture remains fragmented Europe responding to gene therapy challenge, but picture remains fragmented

    Last year saw the launch of three groundbreaking products in Europe: the CAR-T competitors, Kymriah from Novartis and Gilead’s Yescarta and Spark’s rare eye disease gene therapy Luxturna. ... Giorgio Iotti, head of programme leadership and control,

  • Sanofi exits R&D pact with Alnylam on rare disease Sanofi exits R&D pact with Alnylam on rare disease

    Exit also frees up $700m equity investment. Sanofi is bowing out of a five-year-old R&D alliance with Alnylam on rare disease therapies, but the partners will continue to ... Sanofi and Alnylam started working together on gene-silencing therapies in 2014,

  • Pfizer hedges bets on gene therapy deal Pfizer hedges bets on gene therapy deal

    Pfizer's rare disease leader Seng Cheng. “VTX-801 could provide a potentially transformative therapeutic option for patients with Wilson disease by directly addressing the underlying cause of the disease—the ... inability to excrete copper owing to a

  • Sanofi extends growth spurt thanks to Genzyme, Dupixent Sanofi extends growth spurt thanks to Genzyme, Dupixent

    US approval of Cablivi adds to good news. Sanofi has put a long run of decline caused by patent losses in its rear view mirror, with rare disease unit Genzyme and ... There was positive news for Sanofi’s new play in rare blood disorders.

  • Sanofi gets EU OK for Ablynx flagship drug Cablivi Sanofi gets EU OK for Ablynx flagship drug Cablivi

    The new drug is the first new product to come from Sanofi’s push into rare disease therapies and specifically rare blood disorders, which was also behind its $11.6bn acquisition ... s Genzyme rare disease unit.

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  • Focus on Rare Diseases

    Alison Cantle, Rare Disease Business Unit Director at Lucid Group, explains what this means for medical education agencies: “As pharmaceutical organisations focus on personalised medicine, the need for rare disease expertise ... Within this unit, Lucid

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