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rare diseases

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Clinigen launches educational programme for rare disease patients and organisations

The NaviGATE programme will aim to empower patients and provide navigational support

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AstraZeneca’s rare disease unit partners with Verge Genomics in deal worth over $840m

Both will identify drug targets for neurodegenerative and neuromuscular diseases

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AstraZeneca shares positive phase 3 results for Tagrisso combination in lung cancer

An estimated 2.2 million people worldwide are diagnosed with lung cancer each year

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AstraZeneca latest to sue US government over Medicare drug price negotiation plan

The programme already faces court challenges from drugmakers and industry groups

regeneron headquarters

Regeneron’s Veopoz granted FDA approval for ultra-rare immune disease

Fewer than ten patients in the US have been diagnosed with CHAPLE disease

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

Beyfortus will be available in the US ahead of the upcoming RSV season

Biogen Idec building

Biogen to acquire Reata for approximately $7.3bn

The deal gives Biogen access to a recently launched therapy for Friedreich's ataxia

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AstraZeneca’s rare disease unit to acquire Pfizer’s early-stage gene therapies for $1bn

The transaction could potentially see staff associated with the portfolio move to Alexion

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Alliance for Genomic Discovery announces its five founding biopharma members

The AGD aims to accelerate drug development and expand the diversity of genomic data

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AstraZeneca/Sanofi’s RSV antibody receives FDA approval for use in infants

Beyfortus is the now first preventive option approved in the US to protect a broad infant population

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LifeArc launches rare disease programme with £100m investment

An initial £40m will go towards the creation of up to five rare disease research centres

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Horizon shares positive results for Uplizna in neuromyelitis optica spectrum disorder

The rare and debilitating disease affects approximately one to ten per 100,000 people

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