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Compassionate use programme

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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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EMA’s PRIME scheme launches three major new features

The scheme supports the development of treatments targeting unmet medical needs in the EU

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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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BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma

This type of cancer is the most common one diagnosed in teenagers

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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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Turn payer insights into impact with omnichannel engagement in market access

By Betty Charleshouse

Amiculum

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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

Photo of Tim Wright plus title of talk

MFN Pricing Is Moving Fast – Here’s What Matters

With GENEROUS, GUARD, and GLOBE all released since December, 185 pages of policy will materially impact U.S. drug pricing — and yes, it's a lot to digest. In a new...

Genesis Research Group

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ISPOR’s 2026–2027 HEOR Trends report: The Era of Evidence Judgment

In response to ISPOR’s recently published 2026-2027 HEOR Trends, David Miller, CEO, Genesis Research Group, suggests that we’re no longer being evaluated on our ability to produce data, but on...

Genesis Research Group

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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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