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- PMLiVE

Hunter syndrome

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MHRA approves TMC Pharma’s Nulibry to treat rare genetic disease MoCD type A

Molybdenum cofactor deficiency is estimated to affect one in 100,000 to 200,000 newborns worldwide

- PMLiVE

Ionis shares positive late-stage results for olezarsen in familial chylomicronaemia syndrome

The rare genetic disease is estimated to affect up to 13 people per million in the US

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FDA approves Orchard’s Lenmeldy gene therapy for metachromatic leukodystrophy

The rare genetic disease is estimated to affect one in every 40,000 people in the US

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Sanofi presents positive results for Pompe disease enzyme replacement therapy

The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK

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Regenxbio’s investigational gene therapy shows promise in Hunter syndrome

The rare genetic disease is estimated to affect one in every 100,000 to 170,000 births

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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

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AbbVie signs gene therapy deal in eye care

AbbVie and REGENXBIO have teamed up on RGX-314, a potential one-time gene therapy against two leading causes of blindness, wet AMD and diabetic retinopathy.

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

Why personalised medicine based on our own genomes is the future of healthcare

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Shire suffers pipeline failure as takeover speculation builds

Several big pharma companies are reportedly considering bids

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First patient treated with Sangamo’s gene-editing tech

SB-913 uses ZFN technology to insert a corrective gene into the liver cells DNA location

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Shire launches digital ‘paper planes’ for rare disease campaign

#FlyForMPS allows supporters to track the miles their plane travels

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