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- PMLiVE

Compassionate use programme

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

- PMLiVE

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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EU establishes €100m funding scheme for projects tackling public health threats

The HERA Invest fund will focus on health emergency prevention and preparedness initiatives

- PMLiVE

Article: War in the Blood – The battle takes shape in the burgeoning European oncology CAR-T market access space

Tania Rodrigues investigates the market access and commercialization potential for manufacturers of CAR-T therapies in the latest issue of pharmaphorum's Deep Dive.

Research Partnership

- PMLiVE

LifeArc launches rare disease programme with £100m investment

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

- PMLiVE

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

- PMLiVE

NIHR and MRC establish UK Rare Disease Research Platform with £14m investment

The platform brings together expertise from across the UK rare disease research system

- PMLiVE

Research Partnership launches Access CGT

Research Partnership’s specialist market access team have launched Access CGT, a new syndicated access and policy tracker providing surveillance of the cell and gene therapy space.Read more

Research Partnership

- PMLiVE

FDA approves UCB’s Rystiggo to treat generalised myasthenia gravis

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

- PMLiVE

Archetypes: Rethinking go-to-market expectations to drive commercial success

In this white paper, our consultants analyze trends spanning global policy developments, rising inflation, and increasingly complex customer journeys, and reveal a new data-driven approach to archetyping that crystalizes the...

Avalere Health

- PMLiVE

Oncology eBook: Reducing the care gap in cancer prevention, diagnosis, and treatment

Despite significant innovation in the oncology landscape, there is a growing care gap preventing patients accessing and benefiting from innovations in cancer treatment. In this new publication, our oncology experts...

Avalere Health

- PMLiVE

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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