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Bespok Gene Therapy Consortium

- 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

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

Women Leaders Rewriting the Protocols of Rare Disease Care

The true measure of a medical leader is the courage to act when existing systems fail the patient. This is the professional and personal ethos of clinical immunologist Dr. Hilary...

Medscape Education

- PMLiVE

Medscape Wins Anthem Award for Rare Disease Education Platform

Platform has reached 2.4 million healthcare professionals worldwide since 2021. Newark, NJ - [November 18, 2025] - Medscape, the leading digital platform for physicians and healthcare professionals (HCPs), has been...

Medscape Education

- 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

GSK’s Blenrep approved by FDA for multiple myeloma

Approval supported by positive results from phase 3 trial

- PMLiVE

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

- PMLiVE

Roche’s small cell lung cancer therapy approved by FDA

This is the first and only approved combination therapy for extensive-stage small cell lung cancer

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