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Biogen’s salanersen gets FDA Breakthrough Therapy Designation for SMA

In the study, there were improvements in motor function as well as a slowing of neurodegeneration

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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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Biogen agrees $850m felzartamab rights deal with TJ Biopharma

The agreement gives Biogen full global rights to the investigational antibody across immune-mediated diseases

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MHRA authorises Biogen’s higher-dose Spinraza for SMA treatment

SMA is a rare genetic neuromuscular condition affecting around 1,600 people in the UK

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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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Biogen to acquire Apellis in $5.6bn deal

The deal will enhance Biogen’s growth portfolio in immunology and rare disease

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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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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

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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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Biogen’s litifilimab receives FDA Breakthrough Therapy Designation for lupus

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

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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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