RGX-121
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
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
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
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
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
Genomics England receives government funding for cutting-edge genomics research
£105m will be used to accelerate the diagnosis of rare genetic diseases in newborns
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.
Rare genetic diseases – what’s in store for 2021?
Why personalised medicine based on our own genomes is the future of healthcare
Shire suffers pipeline failure as takeover speculation builds
Several big pharma companies are reportedly considering bids
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
Shire launches digital ‘paper planes’ for rare disease campaign
#FlyForMPS allows supporters to track the miles their plane travels