NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug
Pharmaceutical Technology
OCTOBER 17, 2023
The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.
tag paediatrics 
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies
Pharmaceutical Technology
SEPTEMBER 30, 2022
Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.
Developing point-of-care CAR T manufacturing
European Pharmaceutical Review
DECEMBER 27, 2023
Important reassurance about CAR T-cell therapy came with the real-world data that emerged from both large single centres and various consortiums, which included data from both adult and paediatric patients in Israel and Europe. This is in addition to its considerable price tag.
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