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Browsing Tag

rare diseases

8 posts
HHeadlines
Only four people in Australia have been diagnosed with rare genetic disease CHOPS
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Only four people in Australia have been diagnosed with rare genetic disease CHOPS

  • October 11, 2025
Isla Steed is your fairly typical teenager, according to her mum. The 14-year-old is “a stubborn little person”…
HHeadlines
Experimental treatment restores sight to man with genetic eye disorder in Italy
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Experimental treatment restores sight to man with genetic eye disorder in Italy

  • July 30, 2025
By&nbspEuronews Published on 30/07/2025 – 13:02 GMT+2 ADVERTISEMENT In a world first, a man suffering from vision loss…
TTop stories
FDA clears way for Sarepta Therapeutics to use Duchenne drug in some patients
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FDA clears way for Sarepta Therapeutics to use Duchenne drug in some patients

  • July 29, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
TTop stories
Sarepta Therapeutics' Elevidys faces 'arduous' path back to market
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Sarepta Therapeutics’ Elevidys faces ‘arduous’ path back to market

  • July 22, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
TTop stories
Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move
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Sarepta will stop shipping Elevidys, Duchenne gene therapy

  • July 22, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
TTop stories
Sarepta Therapeutics’ Duchenne therapy crisis has a clear lesson
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Sarepta Therapeutics’ Duchenne therapy crisis has a clear lesson

  • July 21, 2025
In a matter of a few days last week, Sarepta Therapeutics announced major layoffs, acknowledged the death of…
TTop stories
Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move
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Sarepta Therapeutics crisis is huge blow to Duchenne families, company

  • July 18, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
TTop stories
Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move
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Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move

  • July 17, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
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