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Sanofi gains rights to Fulcrum’s muscular dystrophy treatment in deal worth more than $1bn

Losmapimod is being investigated as a potential transformative therapy for the rare disease

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Biogen and Eisai announce rolling FDA submission for injectable version of Alzheimer’s drug

The Leqembi SC autoinjector could reduce the need for hospital visits and nursing care compared to IV administration

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Roche announces FDA approval of self-collection solution for HPV screening

More than 13,000 people are diagnosed with cervical cancer every year in the US

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Trial reveals promising results for radiotherapy/hormone therapy combination in prostate cancer

Further evidence is being sought from the phase 2 TRAP trial results through the larger STAR-SHIP study

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Pioneering Paths: Women Leadership in Rare Disease

Rare diseases are often thought of as affecting one in 100,000 or one in 1,000,000 people, and that is true for some conditions. But there are many rare diseases that...

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Takeda gains exclusive option for AC Immune’s Alzheimer’s drug candidate in deal worth over $2.1bn

The agreement includes an option to licence global rights to the anti-amyloid beta immunotherapy

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Sanofi, Pfizer and AstraZeneca commit €2.5bn investment to biomanufacturing projects in France

The investments are part of the wider €15bn investment for the Choose France event

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Novo Nordisk’s haemophilia A therapy Mim8 shows promise in late-stage study

Haemophilia A accounts for up to 85% of the 1,125,000 global haemophilia cases

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WHO opens application period for 2025 Model Lists of Essential Medicines

The deadline to submit ahead of the 25th WHO Expert Committee meeting is 1 November 2024

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Pfizer’s Vyndaqel recommended by NICE to treat rare heart disease ATTR-CM

Approximately 1,500 people in England are affected by transthyretin amyloidosis cardiomyopathy

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PharmaTimes Medical & Scientific Excellence Awards and Nursing Industry Excellence Awards open for entry

Both awards are open for entry until 17 May 2024 and the live awards ceremony will take place on 11 July 2024

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Valneva’s single-dose chikungunya vaccine shows promise in adolescents

The mosquito-borne viral disease often causes sudden, large outbreaks with high attack rates

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