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ALSAwareness

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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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AL-S Pharma release new data from AP-101 phase 2 trial for ALS

ALS is a progressive neurodegenerative disease that affects the motor neurons of the brain and spinal cord

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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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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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Exhibition Trends 2026: What Pharma Exhibitors Need to Know Now

Exhibitions remain one of the most powerful ways for pharmaceutical and life sciences organisations to connect, educate and build trust. Whether you're launching innovations, strengthening partnerships or showcasing research, the...

Apex Events and Exhibitions

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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

Biogen Idec building

Biogen’s Qalsody granted MHRA approval to treat rare form of motor neurone disease

Mutations in the SOD1 gene are responsible for approximately 2% of all cases of the disease

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