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Castleman’s 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

Novartis’ ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease

Approximately half of those with Sjögren’s disease are thought to be undiagnosed

- 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

- 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

FDA launches TEMPO digital health pilot

Digital technologies can improve remote patient care for those with chronic diseases

- PMLiVE

Richard Pazdur, FDA’s drug evaluation chief, to leave FDA just weeks into new role

Pazdur was previously head of the FDA’s Oncology Center of Excellence

- PMLiVE

Merck KGaA given FDA Fast Track designation for generalised myasthenia gravis drug

Cladribine capsules could become the first ever oral treatment for gMG

- PMLiVE

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

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