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dermatology

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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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Matt Ferriday announced as General Manager for LEO Pharma UK

Ferriday most recently held senior leadership roles at Ipsen Biopharma

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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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Almirall celebrates new offices in China to advance innovation in dermatology

The company’s R&D and commercial expertise represent valuable capabilities for future Chinese partners

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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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LEO Pharma’s Anzupgo cream progresses to phase 3 trial for lichen sclerosus

There are currently no approved treatments specifically indicated for LS in the US or Europe

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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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DEBRA Research and University Medical Center Groningen launch clinical trial site for epidermolysis bullosa

This rare genetic disorder causes skin to become extremely fragile

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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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