Health Awareness
Rare diseases 2026
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mRNA therapies ‘address the underlying genetic cause of disease’
New mRNA technologies are in development that have the potential to make a major impact on the lives of children and adults with two rare life-limiting genetic conditions. During the Covid-19 pandemic, a spotlight was shone on an exciting medical advance: messenger RNA (mRNA) technology, which was credited with speeding up vaccine development. The world’s...
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Finding the right patient is the breakthrough: why precision matters in rare disease awareness
Rare disease innovation keeps advancing. New therapies and new mechanisms mean new hope. But here’s the reality: none of it matters if the people who could benefit are never identified. That’s the gap we need to close. Rare disease symptoms are often non-specific. Conditions get misunderstood. Patients spend years moving between appointments without a clear...
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Why scientific innovation alone does not deliver patient impact
Rare disease innovation matters, but it’s only successful if it has an impact on patients. Cross-sector collaboration can help turn Europe’s complexity into access. Scientific innovation is wonderful, agrees Adam Plich. On its own, it’s simply not enough. In fact, he points out, innovation is worthless if it doesn’t create patient impact. Not enough approved...
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The right strategic partners can open a gateway to Europe and the US
Post-Brexit, UK-based biotechs wanting to supply final products into Europe may benefit from the services of an EU-based Contract Development and Manufacturing Organisation (CDMO). When biotech companies move their cell and gene therapy (CGT) programmes from the lab into clinical development, they may not be considering later-stage challenges. Yet, they’ll save huge amounts of time,...
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The hidden cost of PBC fatigue
This article has been commissioned by Ipsen. Ipsen also provided editorial content for this article. Most of us think we understand tiredness: a long day, a short night, a few too many deadlines. Yet, fatigue is different. For many people living with the rare liver disease Primary Biliary Cholangitis (PBC), the exhaustion never fades, no...
