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- PMLiVE

rare genetic diseases

- PMLiVE

Takeda’s Takhzyro granted MHRA approval for younger hereditary angioedema patients

The rare genetic disorder is estimated to affect one in every 50,000 people

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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

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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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MHRA approves Advanz Pharma’s combined antibiotic to treat complicated infections

In England, approximately 130,000 patients are affected by bloodstream infections every year

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Takeda’s HyQvia receives MHRA approval for rare neurological disorder CIDP

The therapy offers patients with CIDP a typical dosing interval of four weeks

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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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Daiichi Sankyo’s Vanflyta approved by MHRA to treat acute myeloid leukaemia

The blood cancer affects approximately 3,100 people in the UK annually

- PMLiVE

Pfizer’s Velsipity receives MHRA and NICE authorisations to treat ulcerative colitis

The immune-mediated inflammatory bowel disease affects approximately 300,000 people in the UK

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MHRA approves UCB’s Rystiggo to treat generalised myasthenia gravis

The autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

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MHRA launches second phase of genetic biobank for stroke prevention medicines

The Yellow Card Biobank will begin investigating direct oral anticoagulants such as apixaban

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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

- PMLiVE

Pfizer/BioNTech’s COVID-19 vaccine available to buy in UK following MHRA endorsement

The XBB.1.5-adapted vaccine will be available for purchase from March 2024

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