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Browsing Tag

genetic disorder

7 posts
HHealth
Enterprise Therapeutics publishes results of Phase 1 study of ETD001, a novel inhaled ENaC blocker for treatment of Cystic Fibrosis, in The Journal of Cystic Fibrosis
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Updated newborn screening guidelines aim to improve cystic fibrosis detection

  • February 2, 2026
An updated report on cystic fibrosis (CF) from Ann & Robert H. Lurie Children’s Hospital of Chicago, in…
HHealthcare
Experimental gene therapy successfully treats dozens of children with rare fatal disorder
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Experimental gene therapy successfully treats dozens of children with rare fatal disorder

  • October 17, 2025
ADVERTISEMENT An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder,…
GGenetics
Pacbio’s Puretarget simplifies carrier screening
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Pacbio’s Puretarget simplifies carrier screening

  • September 30, 2025
Up to 71% of people carry at least one pathogenic variant that could contribute to development of a…
GGenetics
Harmony Biosciences' Genetic Disorder Drug Disappoints In Phase 3 Study
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Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study

  • September 26, 2025
Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results from its Phase 3 registrational clinical trial (the RECONNECT Study)…
GGenetics
Harmony Biosciences' Genetic Disorder Drug Disappoints In Phase 3 Study
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Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study

  • September 24, 2025
Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results…
GGenetics
Genetic testing pilot aims to catch a silent killer
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Genetic testing pilot aims to catch a silent killer

  • September 24, 2025
FH Education Message A potentially life-saving pilot program is launching in Bloomington, Illinois to…
GGenetics
Saol Therapeutics hit with CRL for rare genetic disease
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Saol Therapeutics hit with CRL for rare genetic disease

  • September 10, 2025
The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s…
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