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multicentric Castleman’s disease

- PMLiVE

J&J’s nipocalimab gets FDA Fast Track designation for lupus

The autoimmune disease affects approximately three to five million people globally

- 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

Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma

Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable

- PMLiVE

Johnson & Johnson’s Rybrevant shows positive effects in difficult-to-treat colorectal cancer

Colorectal cancer accounts for around 10% of all cancer cases globally, with an increase in diagnoses in younger people

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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

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AstraZeneca announces $2bn investment in US manufacturing

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

- PMLiVE

J&J receives positive NICE recommendation for multiple myeloma treatment

The treatment is approved for use within the NHS in England and Wales

- 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

Johnson & Jonson secures FDA approval for multiple myeloma treatment

Support for first and only high-risk smouldering multiple myeloma treatment came from positive phase 3 trial results

- PMLiVE

Johnson & Johnson seeks first EMA approval for icotrokinra for plaque psoriasis

Icotrokinra is a first-in-class, once-daily tablet for moderate-to-severe plaque psoriasis

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