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Basliea

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LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

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Astellas’ application to EMA validated for treatment of muscle-invasive bladder cancer

Bladder cancer affects over 224,000 people annually in Europe, with over 30% of cases being muscle-invasive bladder cancer

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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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

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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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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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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Insights From ISPOR 2025: Breaking the Data Barrier in Rare Diseases – Can AI Bridge the Gap?

Last month, the ISPOR annual meeting in Montreal was brimming with opportunities to explore the latest developments and insights in health economics and outcomes research (HEOR) and market access. In...

Petauri Evidence

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Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD

Immunoglobulin G4-related disease affects approximately 20,000 people in the US

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