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Browsing Tag

Rare Diseases

9 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”…
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 25, 2025
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
GGenetics
The genetic answer to rare diseases
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The genetic answer to rare diseases

  • September 13, 2025
The standard treatment is a liver transplant, but this entails a high risk of complications in infants. Thanks…
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

  • September 7, 2025
For the first time, scientists say they have reached into the genome of a severely ill child and…
HHealth
World's first gene therapy for p47 Chronic Granulomatous Disease developed by UCL and GOSH
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World’s first gene therapy for p47 Chronic Granulomatous Disease developed by UCL and GOSH

  • August 21, 2025
image: ©UCL UCL and Great Ormond Street Hospital have developed the world’s first gene therapy for p47 Chronic…
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…
HHealth
Scientists find way to reverse nerve damage in rare ALS form
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Scientists find way to reverse nerve damage in rare ALS form

  • August 8, 2025
image: ©Rost-9D | iStock Researchers using patient-derived stem cells reveal a rare ALS mutation triggers a chronic stress…
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