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Beta thalassaemia

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Ipsen’s Bylvay approved by FDA for severe itching due to Alagille syndrome

As many as 88% of patients living with the condition experience severe pruritus

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Merck’s efinopegdutide shows promise in nonalcoholic fatty liver disease

The candidate was recently granted FDA fast track designation for a severe form of NAFLD

Biogen Idec building

Biogen/Eisai’s Alzheimer’s drug backed by FDA advisory committee for traditional approval

Leqembi was granted accelerated approval by the FDA in January for patients with early AD

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AstraZeneca/Sanofi’s RSV antibody backed by FDA advisory committee for infants

RSV is the leading cause of hospitalisation for infants aged under one year in the US

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Pfizer’s RSV vaccine approved by FDA for older adults

RSV infections in older adults account for up to 160,000 hospitalisations each year in the US

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FDA approves Blueprint Medicines’ Ayvakit for indolent systemic mastocytosis

Ayvakit is now the first and only approved medicine designed to treat the underlying cause of the disease

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Decision date for Sarepta’s Duchenne muscular dystrophy gene therapy delayed by FDA

The disease occurs in up to one in every 5,000 newborn males worldwide

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AbbVie’s Rinvoq receives FDA approval for Crohn’s disease

Inflammatory bowel disease affects nearly one in every 100 people in the US

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Pfizer’s maternal RSV vaccine candidate backed by FDA advisory committee

Up to 80,000 children aged under five are hospitalised each year in the US due to RSV infection

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AbbVie and Genmab’s blood cancer therapy granted FDA accelerated approval

Approximately 150,000 new cases of DLBCL are diagnosed globally each year

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FDA advisory committee backs Sarepta’s Duchenne muscular dystrophy gene therapy

The disease occurs in up to one in every 5,000 newborn males worldwide

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Astellas Pharma’s non-hormonal menopause drug granted FDA approval

Vasomotor symptoms affect up to 80% of menopausal individuals in the US

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