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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.

GSK sells eczema and psoriasis drug to Dermavant for £250m

GSK sells eczema and psoriasis drug to Dermavant for £250m

GSK’s tighter focus on respiratory, HIV, oncology, and vaccines has already seen it cull 30 pipeline programmes and offload its rare disease assets to Orchard Therapeutics. ... Those are for the more severe end of the symptom spectrum, but there’s

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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

  • 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

  • Mereo BioPharma appoints head of patient access and commercial planning Mereo BioPharma appoints head of patient access and commercial planning

    rare disease commercial infrastructure. ... In this role, Hughes-Wilson was responsible for Sobi’s commercialisation approach and led pricing, reimbursement and access teams for the group’s rare disease product portfolio.

  • Amryt Pharma appoints new regulatory affairs head Amryt Pharma appoints new regulatory affairs head

    Amryt Pharma appoints new regulatory affairs head. Derval O’Carroll joins the rare disease-focused biopharma from Retrophin. ... Prior to joining Amryt, she served as senior director of regulatory affairs for rare disease group Retrophin.

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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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