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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.

Gilead NASH drug clears mid-stage trial, but rivals are looming

Gilead NASH drug clears mid-stage trial, but rivals are looming

All Pfizer’s drugs are in early- to mid-stage development. Orphan disease. ... Meanwhile, Gilead also reported phase 2 results with GS-9674 in another liver condition, a rare disease called sclerosing cholangitis that causes the network of ducts that

Latest news

  • BioMarin eyes 2019 FDA filing for haemophilia A gene therapy BioMarin eyes 2019 FDA filing for haemophilia A gene therapy

    Rare disease specialist BioMarin is hoping to file for approval its haemophilia A gene therapy valoctocogene roxaparvovec (valrox) in the second half of next year, if its ongoing phase 1/2 ... short limbs) seen with the disease.

  • Orchard surpasses IPO expectations, raising $200m Orchard surpasses IPO expectations, raising $200m

    Orchard surpasses IPO expectations, raising $200m. Funds will progress rare disease portfolio. ... Central to its plans is to further develop its newly acquired rare disease portfolio from GlaxoSmithKline (GSK).

  • Dupixent helps put Sanofi back on growth track Dupixent helps put Sanofi back on growth track

    Gains for vaccine division Sanofi Pasteur and the company’s specialty medicines division, which along with Dupixent includes Genzyme’s rare disease drugs and multiple sclerosis therapies like Aubagio (teriflunomide), contributed

  • Shire’s angioedema drug Takhzyro recommended in Europe Shire’s angioedema drug Takhzyro recommended in Europe

    The European Medicines Agency's CHMP committee has recommended Shire’s Takhzyro (lanadelumab), a new rare disease treatment which has blockbuster potential. ... We are excited about the future potential of lanadelumab in helping to address the needs of

  • Encouraging data for GenSight’s blinding retinal disease gene therapy Encouraging data for GenSight’s blinding retinal disease gene therapy

    Encouraging data for GenSight’ s blinding retinal disease gene therapy. Better results after April's setback. ... Gensight has revealed some promising data for its gene therapy GS010, which is currently in testing to treat those with Leber hereditary

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Latest Intelligence

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in number and geographically spread,

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Accelerating diagnosis in rare disease. Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in

  • Significant figures Significant figures

    ALIS (Amikacin liposome inhalation suspension) is being developed as a treatment for refractory non tuberculous Mycobacterial (NTM) lung disease, a rare progressive, destructive infection caused by mycobacterium avium complex. ... NTM is a rare

  • Deal Watch January 2018

    Rare blood disease products in vogue - Sanofi part 2. In the early part of January it was made public that Novo Nordisk had made an unsolicited $3.1bn (28 per share) ... Sanofi was also interested in caplacizumab, which fits not only with its rare

  • The real-world data conundrum The real-world data conundrum

    promise around treating rare diseases. ... However, there is still high unmet need: fewer than one in ten patients with a rare condition receive disease-specific treatment and approved medicines are available for only 5% of rare

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Latest appointments

  • Ipsen hires general manager, Laura Hamill joins Gilead and Orchard expands leadership team Ipsen hires general manager, Laura Hamill joins Gilead and Orchard expands leadership team

    expertise as we shape our business for future growth with an unrelenting commitment to enhancing the lives of patients living with rare disease.

  • BMS appoints CCO, Stephen Davies joins Eisai and more BMS appoints CCO, Stephen Davies joins Eisai and more

    Sobi appoints head of technical operations. Rare disease specialist company Sobi has appointed Anne Marie de Jonge Schuermans to front its new technical operations organisation. ... Commenting on her appointment, Schuermans said: “Sobi has unique

  • Abeona Therapeutics names CEO Abeona Therapeutics names CEO

    Rare genetic disease focused biopharma Abeona Therapeutics has named its chief executive officer in the form of Carsten Thiel. ... Steven Rouhandeh, executive chairman of Abeona, said: “As CEO, Carsten Thiel brings a unique combination of extensive

  • Therachon poaches Shire’s Aled Williams Therachon poaches Shire’s Aled Williams

    Williams has more than 25 years’experience in healthcare and pharmaceuticals and most recently he served as vice president and global strategy head for rare gastrointestinal diseases at Shire. ... Commenting on the recent appointment, Luca Santarelli,

  • Open Health creates new role Open Health creates new role

    Open Health creates new role. Gavin Jones joins the agency as its director of rare disease. ... In order to strengthen their experience and expertise, healthcare communications and market access group Open Health has appointed Gavin Jones into its newly

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