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Browsing Tag

rare diseases

13 posts
GGenetics
GeneDx Announced as Sequencing Partner for Sunshine Genetics Act Pilot
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GeneDx Announced as Sequencing Partner for Sunshine Genetics Act Pilot

  • October 15, 2025
Source: JGI/Jamie Grill / Getty Images Genetic sequencing and diagnostics company GeneDx announced this week that it would…
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
NLRP3 inflammasome and hearing loss: from mechanisms to therapies | Journal of Neuroinflammation
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NLRP3 inflammasome and hearing loss: from mechanisms to therapies | Journal of Neuroinflammation

  • October 6, 2025
Sensorineural hearing loss (SNHL) affects approximately 1.5 billion people globally. Approximately 430 million of these patients require rehabilitation…
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 5, 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
Developments in diagnostic and surgical techniques in children with sagittal suture craniosynostosis: a systematic review spanning the last 30 years | Orphanet Journal of Rare Diseases
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Developments in diagnostic and surgical techniques in children with sagittal suture craniosynostosis: a systematic review spanning the last 30 years | Orphanet Journal of Rare Diseases

  • August 17, 2025
Literature search and study selection An initial search using the search terms related to the operative technique yielded…
MMarkets
Global Pharmaceutical Market Projected to Hit $2.2 Trillion by 2029
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Global Pharmaceutical Market Projected to Hit $2.2 Trillion by 2029

  • July 22, 2025
BCC Research LLC “Driven by Innovation in Biologics and Expanding Therapeutic Applications, the Global Pharmaceutical Drugs Market Continues…
NNews
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…
BBusiness
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…
United States
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