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New NHS treatments increase survival rates for babies with spinal muscular atrophy

Around 70 babies are born with the rare muscle-wasting disease each year in the UK

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

- PMLiVE

NHS extends eligibility for shingles vaccine to almost one million people

Approximately one in four people will develop shingles and the risk increases with age

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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

- 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

Bristol Myers Squibb’s Sotyktu recommended by NICE for plaque psoriasis

An estimated 1.8 million people in the UK are affected by some form of the condition

- 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

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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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NHS to introduce world-first genetic blood-matching test for transfusion treatments

NHS Blood and Transplant will receive almost £1m in funding from NHS England

- PMLiVE

NHS spring COVID-19 booster programme opens up for those at highest risk

Around five million people are eligible for the booster in line with the JCVI’s advice

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