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FDA approves Italfarmaco’s Duvyzat as first nonsteroidal Duchenne muscular dystrophy drug

The neuromuscular genetic disease is estimated to affect one in every 3,500 to 6,000 male births worldwide

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Takeda’s Iclusig combination granted FDA accelerated approval for rare form of leukaemia

The drug is now the first targeted therapy approved as a frontline treatment for Ph-positive ALL

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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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FDA approves Madrigal’s Rezdiffra as first drug for fatty liver disease NASH

Over six million people in the US are affected by NASH with moderate-to-advanced liver scarring

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Bristol Myers Squibb’s Breyanzi granted FDA accelerated approval for non-Hodgkin lymphomas

About 20,700 new cases of chronic lymphocytic leukaemia are expected to be diagnosed in the US this year

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BeiGene’s Tevimbra receives FDA approval to treat advanced oesophageal cancer

More than two-thirds of patients have advanced or metastatic disease at the time of diagnosis

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Pfizer’s 20-valent pneumococcal vaccine receives EC approval for infants and children

The 20 serotypes included in Prevenar 20 cause the majority of circulating pneumococcal disease

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FDA approves Mirum’s Livmarli to treat cholestatic pruritus in rare liver disease patients

Progressive familial intrahepatic cholestasis affects one in every 50,000 to 100,000 births in the US and Europe

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Pfizer’s Adcetris combination shows promise in phase 3 large B-cell lymphoma study

The aggressive blood cancer affects more than 25,000 people in the US every year

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Regeneron’s Praluent receives FDA approval for paediatric patients with genetic high cholesterol

Patients aged eight year or older with heterozygous familial hypercholesterolaemia will be eligible to receive the treatment

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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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Bristol Myers Squibb’s Opdivo combination receives FDA approval for urothelial carcinoma

More than 83,000 news cases of bladder cancer are expected to be diagnosed in the US this year

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