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- PMLiVE

Fabry disease

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AbbVie announces NICE recommendation for blood cancer therapy Tepkinly

Around 5,500 new cases of diffuse large B-cell lymphoma are diagnosed in the UK every year

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NICE recommends Chiesi’s alpha-mannosidosis enzyme replacement therapy

Alpha-mannosidosis, an extremely rare inherited condition characterised by a broad range of impairments, affects approximately 25 people in England

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NIHR awards £2.3m for AI study to improve radiotherapy for lung cancer

The AI tool is expected to help doctors plan each patient’s radiotherapy treatment

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Bristol Myers Squibb’s Camzyos recommended by NICE for inherited heart condition

One in 500 people in the UK are affected by hypertrophic cardiomyopathy

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NICE recommends Chiesi’s Elfabrio for adults with Fabry disease

Affecting approximately 1,150 people in England, Fabry disease is a rare genetic disease that can lead to progressive damage to vital organs

- PMLiVE

NICE recommends AI technologies for radiotherapy treatment planning

AI technology could provide the NHS with significant time and cost benefits

Patient Centric Approaches for Rare and Ultra-Rare Diseases

Lawrence Bressler, Head of Value & Market Access, Global Rare Diseases at Chiesi, shares how Chiesi is revolutionizing the lives of people living with rare diseases on a global scale....

Impetus Digital

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Protalix, Chiesi’s monthly treatment PRX-102 shows benefit in Fabry disease

Fabry disease is a rare disorder in which patients inherit a deficiency of the α–Galactosidase–A enzyme

- PMLiVE

Moderna inks R&D agreements with Vertex and Chiesi

Biotech agrees to separate deals for the development of gene-editing and mRNA therapies

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Chiesi launches new rare disease R&D division

New business unit will be based in Boston, US

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NICE backs Europe’s first stem cell therapy

Holoclar is approved for routine use in patients with LSCD

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Amicus Therapeutics launches Fabry treatment Galafold in Italy

Follows publication of Ministry of Health's reimbursement guidelines

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