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- PMLiVE

UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

- PMLiVE

Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD

Around 1.4 million people in the UK are diagnosed with COPD, with two million thought to be undiagnosed

- PMLiVE

AbbVie reaches agreement for affordable prescription drugs with US government

The pharma company has also pledged a $100bn investment in US infrastructure over the next decade

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

- PMLiVE

AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

- PMLiVE

ViiV Healthcare receives NICE recommendation for HIV prevention therapy

The treatment is the first and only long-acting HIV prevention option available on the NHS in England and Wales

- PMLiVE

Pfizer’s Lorviqua recommended by NICE for lung cancer

Lung cancer is the leading cause of cancer-related deaths worldwide

- PMLiVE

AbbVie/Genmab’s lymphoma drug shows promise in phase 3 combination trial

Follicular lymphoma affects around 15,000 people in the US every year

- PMLiVE

AbbVie shares promising phase 3 results for Rinvoq in severe alopecia areata

Around 700,000 people in the US are living with some form of the autoimmune disease

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