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Raptor Pharmaceuticals

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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

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

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

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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FDA launches TEMPO digital health pilot

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

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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

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

Cladribine capsules could become the first ever oral treatment for gMG

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AstraZeneca announces $2bn investment in US manufacturing

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

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AstraZeneca reports positive results from rare disease trial

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

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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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GSK’s Blenrep approved by FDA for multiple myeloma

Approval supported by positive results from phase 3 trial

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MHRA and FDA expand collaboration on AI and medtech regulation

National Commission on the Regulation of AI in Healthcare brings together UK and US experts

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