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genetic diseases
This page shows the latest genetic diseases news and features for those working in and with pharma, biotech and healthcare.
UK government announces new rare diseases action plan for England
Data sharing will also be increased to allow for a fuller picture of how people are tested for genetic diseases across England, and it will be made easier for patients with ... Health minister, Helen Whately, said: “Rare diseases are individually rare
Latest news
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Genomics England receives government funding for cutting-edge genomics research
£105m will be used to accelerate the diagnosis of rare genetic diseases in newborns. ... rare genetic diseases and patients with cancer to benefit from earlier diagnosis and faster access to treatment.
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NHS England launches national genetic testing service for babies and children
NHS England has announced the launch of a ‘world-first’ national genetic testing service to deliver rapid life-saving checks for children and babies. ... results within days – meaning they can kick start lifesaving treatment plans for more than 6,
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Merck and Orna agree on deal worth $3.5bn for RNA technology
Orna will retain rights to its oRNA-LNP technology platform and will continue to advance other wholly owned programmes in areas such as oncology and genetic diseases. ... Cerevance, a specialist in diseases affecting the central nervous system, plans to
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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.
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Takeda agrees on $2bn deal with Code Bio to expand its gene therapy programmes
The pharma company will gain opt-in rights for four rare diseases candidates. ... Natarajan added: “We aim to provide functional cures to patients with rare genetic and haematologic diseases through next-generation gene therapy programmes.
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Latest Intelligence
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Gene therapy, gene editing and perspectives on future use in Parkinson’s disease
The application of this technology is clear in monogenic diseases and its potential is now being investigated for idiopathic diseases without a known single genetic cause, such as Parkinson’s disease ... Animal model research is ongoing and greater
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Rare diseases: not so rare after all
Genetic diseases are typically rare, manifest at an early age and progress to a spectrum of debilitating manifestations that put a massive strain on the parents and carers; the effect for ... New approaches. Historically, the small case numbers of rare
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How early-stage clinical research is facilitating the new wave of personalised medicines
It is a shift away from a ‘one-sizefits-all’ approach to treatment to one which uses new approaches, such as identifying genetic and other clinical information, to tailor therapies that ... This type of technology is becoming a reality for diseases
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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
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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.
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Latest appointments
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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.”.
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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.
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US biotech Translate Bio appoints Daniella Beckman
part of a dedicated team focused on developing innovative products for genetic diseases.”.
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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
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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.”.
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Latest from PMHub
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The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia
Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. ... patients and families affected by devastating diseases
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Overcoming Patient Recruitment Challenges in Rare Disease Trials
Rare diseases are thought to affect up to 446 million people worldwide. ... With more than 7, 000 rare diseases, most of which are genetic, it’s vital all patients can access new and effective treatments.
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FREE Live Webinar - Overcoming Patient Recruitment Challenges in Rare Disease Trials
Rare diseases are thought to affect up to 446 million people worldwide. ... With more than 7, 000 rare diseases, most of which are genetic, it’s vital all patients can access new and effective treatments.
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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
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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
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- Pfizer reports positive results for elranatamab in multiple myeloma
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