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Eisai and Biogen’s new data shows Alzheimer’s patients choose to stay on Leqembi long-term

The findings were presented at the 20th International Conference on Alzheimer’s and Parkinson’s Diseases in Denmark

- 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

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Biogen’s litifilimab receives FDA Breakthrough Therapy Designation for lupus

Therapies like topical steroids manage symptoms but there is currently no cure

- PMLiVE

Eisai and Biogen’s subcutaneous Leqembi given FDA Priority Review for early Alzheimer’s

Administering the drug subcutaneously rather than intravenously showed similar clinical benefits for patients

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smartbax licenses compound from Aicuris for next-generation antibiotic development

The new programme addresses the urgent unmet medical need for new therapies to tackle antibiotic-resistant infections

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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

- PMLiVE

Eisai submits new drug application for subcutaneous Leqembi in Japan

LEQEMBI is a treatment for early Alzheimer’s disease

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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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Biogen and Eisai receive MHRA approval for updated dosing of early Alzheimer’s treatment

The MHRA approval for IV dosing comes following positive results from the global phase 3 trial

- PMLiVE

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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