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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.

Shire’s bleeding disorder drug approved in Europe

Shire’s bleeding disorder drug approved in Europe

Shire’ s bleeding disorder drug approved in Europe. Veyvondi for patients with rare disease von Willebrand disease. ... The European Commission (EC) has given the green light to Shire’s haemostatic disorder treatment Veyvondi, which is now set to

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Latest Intelligence

  • The European Medicines Agency: PRIME’d for access? The European Medicines Agency: PRIME’d for access?

    RG6042 (formerly named IONIS-HTTRx) is in development for Huntington’s disease. ... It’s hoped it could be a big step in treatment for the genetically-inherited and rare wasting disease.

  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    pancreatitis. Then GSK’s Strimvelis followed in 2016 for a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). ... However this small molecule drug promises more modest treatment benefits, and

  • 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

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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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Latest from PMHub

  • Medical information in precision medicine

    With new gene therapies, we could even prevent the disease developing in the first place. ... The patient and caregiver populations in the rare disease sector are highly engaged.

  • Biotech: it's a people business

    During our research into the sector, we have found a simple formula for driving success in biotech organisations, which begins with a group of patients, often with a rare disease. ... What is your advice for understanding and addressing the real needs of

  • Raising Awareness: Rare Disease Day

    Initiatives such as Rare Disease Day aim to raise awareness of these unusual disorders. ... Want to find out more? Visit the Rare Disease Day website at: www.rarediseaseday.org.

  • Why I think rare disease companies are more patient-centric

    The company size plays a big role. Rare disease companies are generally smaller, and this can allow individualised focus and dedication. ... Rare disease companies perhaps tend to attract employees who consider themselves too, patient-centric.

  • A Spotlight on Healthcare Communications Advocacy by Alastair Kent

    He is also Chair of Rare Disease UK. Rare Disease UK is a national alliance for people with rare diseases and all who support them. ... Alastair has worked in the field of genetic and rare disease healthcare for over 20 years.

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