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Waldenström’s macroglobulinemia

- 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

NICE recommends ALK’s immunotherapy to target underlying cause of hay fever

An estimated 26,800 people in England with moderate-to-severe cases could benefit from Itulazax

- PMLiVE

NICE recommends BeOne’s Brukinsa to treat mantle cell lymphoma on NHS

Approximately 600 people are affected by the form of non-Hodgkin lymphoma every year in the UK

- PMLiVE

Novartis’ Kisqali recommended by NICE to treat broad early breast cancer population

The drug was previously available only for patients whose cancer had spread to nearby lymph nodes

- PMLiVE

Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE

The rare inherited disease affects approximately 11,000 people in the UK

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CHMP recommends tablet formulation of BeOne’s targeted cancer drug Brukinsa

The new formulation would allow blood cancer patients to halve their daily pill intake

- PMLiVE

Boehringer’s Spevigo recommended by NICE for generalised pustular psoriasis

The rare form of psoriasis can lead to serious complications such as sepsis and heart failure

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