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Rare Disease Innovation Hub

- PMLiVE

LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

- PMLiVE

Shionogi starts phase 2 clinical trial for Pompe Disease

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

- PMLiVE

PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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Pistoia Alliance publishes first best-practice framework for ethical social media use in drug development

The Alliance calls for funding and expertise for the next phase of its social media project, marking its first direct engagement with oncology, rare disease and cardiology patients

- PMLiVE

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

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

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

- PMLiVE

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

- PMLiVE

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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FDA unveils new voucher programme to accelerate drug review process

The programme is designed to reduce the review time for companies supporting US national interests

- PMLiVE

Eli Lilly to expand long-standing Purdue University partnership with $250m investment

The alliance will aim to bring medicines to patients faster using advanced technologies

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