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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.

Hemlibra off to a flyer as Roche raises 2019 forecasts

Hemlibra off to a flyer as Roche raises 2019 forecasts

There are some big filings due this year – including risdiplam for rare disease spinal muscular atrophy that will take on Biogen’s big-selling Spinraza (nusinersen) – and Roche is also hoping

Latest news

  • Akcea steals march on Alnylam with NICE approval Akcea steals march on Alnylam with NICE approval

    Advantage to Tegsedi over Onpattro. NICE has recommended Akcea Therapeutics’ rare disease treatment Tegsedi (inotersen), after the company improved its price offer. ... Richard A. Jones,  SVP Head of Europe for Akcea Therapeutics. “hATTR amyloidosis

  • Alnylam swaps Sanofi for Regeneron as R&D partner Alnylam swaps Sanofi for Regeneron as R&D partner

    The new deal – announced just after Sanofi and Alnylam dissolved a rare disease R&D partnership set up in 2014 – will see Regeneron pay $400m upfront to the RNA interference specialist ... Regeneron will however take the lead on eye disease candidates

  • UniQure gets speedy US review for Huntington’s gene therapy UniQure gets speedy US review for Huntington’s gene therapy

    vector carrying gene designed to reduce levels of an overexpressed protein in Huntington’s disease. ... However the therapy for ultra-rare inherited disease lipoprotein lipase deficiency (LPLD), costing more than 1m per treatment, w as withdrawn five

  • Biogen’s Spinraza overpriced, says ICER Biogen’s Spinraza overpriced, says ICER

    Expensive rare disease drugs like Biogen’s spinal muscular atrophy (SMA) therapy are “threatening the affordability and sustainability of the US health system”, says ICER.

  • Sangamo surges on data for haemophilia A gene therapy Sangamo surges on data for haemophilia A gene therapy

    nuclease (ZFN) gene-editing platform for rare disease Hunter syndrome. ... reduces risk in the company’s other gene therapy programmes which include candidates for Fabry disease, amyotrophic lateral sclerosis and Huntington’s disease.

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Approximately 10 fully matching, plus 407 partially matching documents found.

Latest Intelligence

  • The first, my last, my everything The first, my last, my everything

    In a widespread shift from volume to value, around 80% of molecules in development at present are in oncology or rare disease. ... In the past, industry has taken a traditional view of patient value, typically basing it on clinical outcomes and disease

  • The Orkambi row explained The Orkambi row explained

    To what extent does the row reflect bigger industry/healthcare issues? Orkambi does reflect wider concerns about rare disease drugs that are assessed via NICE’s single technology appraisal (STA) system. ... disease. February 2019: Patient activist

  • #OrkambiNow? #OrkambiNow?

    The STA system doesn’t account for the rarity of a disease in the same way NICE’s Highly Specialised Technology (HST) appraisal for ultra- rare diseases can. ... rare disease campaigners say a fundamental re-think is needed.

  • Genomics integration: can the NHS rise to the challenge? Genomics integration: can the NHS rise to the challenge?

    Last year, NHS England announced that it will focus on earlier diagnosis and more personalised medicines for cancer and rare disease patients, and it will further that goal by launching a ... service. Deans explained: “There are different ways to

  • Rare Diseases Day Rare Diseases Day

    One in 20 people will be affected by a rare disease at some point in their life, according to Eurordis. ... between medical, social and support services in order to tackle the challenges that people living with a rare disease and their families around

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Approximately 2 fully matching, plus 79 partially matching documents found.

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 new commercial leader, Davies joins Eisai and more BMS appoints new commercial leader, 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

    Carsten Thiel joins the biopharma from Alexion. 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

  • 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

    In order to strengthen their experience and expertise, healthcare communications and market access group Open Health has appointed Gavin Jones into its newly formed role of director of rare disease. ... Commenting on his appointments, Sandy Roydon, chief

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Approximately 0 fully matching, plus 30 partially matching documents found.

Latest from PMHub

  • emotive supports Rare Disease Day 2019

    With a series of events and activities over the week leading up to Rare Disease Day on February 28th, we showed our support for stimulating greater awareness of Rare Diseases along ... In line with the Rare Disease Day theme, we took photos of our team

  • Porphyria Awareness Week – Round Up

    Porphyria Awareness Week has given the team at emotive an opportunity to show our support for those touched by this debilitating Rare Disease as well as our enthusiasm for working in ... Porphyria is a rare condition that is thought to affect around 1 in

  • Three amazing women at emotive talk about what they love about medical communications

    Everyone is passionate about their work. Working in rare diseases, where there are so many exciting new advances, I feel good about what we do. ... emotive is an award-winning healthcare communications agency working with leading global life sciences

  • Rewarding excellence within Porterhouse Medical’s scientific services team.

    They have made an excellent start to their careers in medical communications, supporting the wider teams on a number of key activities across the haematology and rare disease teams.

  • Rare Disease Day 2019

    Today is #RareDiseaseDay and is an opportunity to highlight some truly unbelievable/frightening statistics around rare diseases. ... Did you know, for example, that over 3.5 million people in the UK suffer from a rare disease or that 95% of rare diseases

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Approximately 9 fully matching, plus 46 partially matching documents found.

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