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Rare genetic diseases

This page shows the latest Rare genetic diseases news and features for those working in and with pharma, biotech and healthcare.

UK government allocates up to £680m a year to two funds offering cutting-edge treatments

UK government allocates up to £680m a year to two funds offering cutting-edge treatments

The IMF will provide potentially life-saving drugs for rare and genetic diseases, with the UK government pledging up to £680m per year to be divided up between the funds. ... For those living with rare conditions, approving treatments for rare disease

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Latest Intelligence

  • EU cross-border healthcare EU cross-border healthcare

    One truth is certain for all rare diseases, but especially for severe genetic ones: the faster patients get the right treatment, the better their chances of living a full life or ... However, although the EU’s approach to cross- border healthcare is a

  • Rare genetic diseases – what’s in store for 2021? Rare genetic diseases – what’s in store for 2021?

    The cross-collaboration has also brought forward fundamental changes and breakthroughs for the treatment of many rare genetic diseases. ... We may be able to firmly establish genomic profiling to accelerate the diagnosis of rare genetic diseases.

  • Successful product commercialisation: what can we learn from the rare disease setting? Successful product commercialisation: what can we learn from the rare disease setting?

    specialty or orphan drug status – all of which are typically associated with drugs targeting rare diseases. ... Ensuring a holistic approach to patient care. Rare diseases are often genetic, disproportionately affect children, and carry a high burden

  • Staying ahead in the era of precision medicine Staying ahead in the era of precision medicine

    and genetic diseases. ... Empowering and uniting patient advocacy groups to raise awareness around the need to evolve the clinical trial and approval processes, particularly for rare diseases, where the strongest data will only come

  • 25 Women Leaders in UK Healthcare 25 Women Leaders in UK Healthcare

    4. JAYNE SPINK . Rare disease patient champion. Medical research is expanding our understanding of genetics and genetically inherited rare diseases daily, but this progress is agonisingly slow if you are living ... Genetic Alliance UK is home to Rare

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Latest appointments

  • Gamida Cell strengthens leadership team Gamida Cell strengthens leadership team

    Their combined experience in corporate strategy, business development and commercialisation will be invaluable to the company’s continued growth as we advance our clinical programmes in blood cancers and rare genetic ... diseases.”.

  • Ann Barbier joins Translate Bio as its chief medical officer Ann Barbier joins Translate Bio as its chief medical officer

    Prior to joining Translate Bio, Barbier was vice president of clinical development, rare genetic diseases at Agios Pharmaceuticals, where she led the development programme of a small molecule in rare benign ... haematological diseases.

  • Novartis' Owen Wallace to lead Fulcrum Therapeutics Novartis' Owen Wallace to lead Fulcrum Therapeutics

    He said: “I am thrilled to be joining Fulcrum in its efforts to pioneer its bold new approach to modulating gene expression to treat the fundamental causes of rare genetic diseases.”. ... He said: “Fulcrum is uniquely committed to its mission of

  • Therachon appoints new chief medical officer Therachon appoints new chief medical officer

    He will play a key role in guiding TA-46 from discovery into clinical development and helping us fulfill our mission of developing medicines for rare, genetic diseases.”.

  • Therachon strengthens management team Therachon strengthens management team

    Dr Porter (pictured right) becomes chief operating officer at the rare genetic diseases specialist, brings over two decades of industry experience across multiple therapeutic areas. ... Most recently, he was global head of operations management for

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Latest from PMHub

  • Improving healthcare for rare disease patients Improving healthcare for rare disease patients

    With the majority of rare diseases having a genetic component, the more widespread use of whole genome sequencing in recent years has helped with the detection of rare genetic abnormalities and ... Resource.Accessed March 1, 2022. Genetic and Rare

  • Rare Disease Day – looking to the future with hope Rare Disease Day – looking to the future with hope

    by a genetic deficiency in an enzyme involved in tyrosine metabolism, which results in a build-up of homogentisic acid, manifesting in ‘black urine’ ( Orphanet Journal of Rare Diseases). ... diseases. The study demonstrated an increase in diagnostic

  • Rare Disease Day in a COVID-19 environment

    1). With 8 out of 10 rare diseases considered to have a genetic component. ... Clinical Geneticists and Genetic Counsellors are part of the multi-disciplinary team and network of dedicated professionals who ensure continuity of care and treatment for

  • OPEN Health’s virtual reflections on the World Orphan Drug Congress US OPEN Health’s virtual reflections on the World Orphan Drug Congress US

    We learned throughout the week that more than an estimated 40% of future drug approvals will concentrate on rare diseases and genetic disorders. ... Improving the diagnosis of rare diseases is a typical challenge and it was interesting to hear the

  • AMICULUM® launches new specialist agency: AMICULUM Access

    Never has this been more apparent than with the increasing number of exciting products developed for rare and genetic diseases. ... AMICULUM already delivers high-quality market access projects through our medical communications teams – especially in

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