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Browsing Tag

Rare diseases

8 posts
GGenetics
GeneDx to Partner with Florida’s Sunshine Genetics Network, Launching Nation’s First State-Backed Genomic Newborn Screening Program
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GeneDx to Partner with Florida’s Sunshine Genetics Network, Launching Nation’s First State-Backed Genomic Newborn Screening Program

  • October 13, 2025
Florida Institute for Pediatric Rare Diseases and Representative Adam Anderson ushering in a new era of preventative care…
GGenetics
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”…
HHealthcare
Coverage of Sarepta Duchenne drug may be halted by N.Y. state Medicaid
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Coverage of Sarepta Duchenne drug may be halted by N.Y. state Medicaid

  • October 8, 2025
In a setback for Sarepta Therapeutics, a New York panel has voted unanimously to recommend the state Medicaid…
GGenetics
Knowing patient’s genetic make-up may change the way medicines are prescribed
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Knowing patient’s genetic make-up may change the way medicines are prescribed

  • October 4, 2025
SINGAPORE – Not all medicines work the same way on every patient, and part of the answer lies…
GGenetics
Acadia Pharmaceuticals drug for rare genetic condition fails late-stage study
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Acadia Pharmaceuticals drug for rare genetic condition fails late-stage study

  • September 24, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
GGenetics
Birmingham Live
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More than 1,000 women sign up for vital Birmingham study to detect rare genetic diseases

  • August 30, 2025
Hundreds of women have stepped up to help a Birmingham hospital detect rare genetic diseases. More than 1,200…
GGenetics
CRISPR used in landmark treatment to fix misspelling in baby's genome
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CRISPR used in landmark treatment to fix misspelling in baby’s genome

  • August 26, 2025
For the first time, scientists say they have reached into the genome of a severely ill child and…
HHealthcare
US drug pricing shake-up threatens access to medicines in Europe – POLITICO
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US drug pricing shake-up threatens access to medicines in Europe – POLITICO

  • August 20, 2025
For European drugmakers there are still many unknowns about how the MFN scheme — and overall tariffs on…
Ireland
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