Experimental gene therapy successfully treats dozens of children with rare fatal disorder
ADVERTISEMENT An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder,…
Pacbio’s Puretarget simplifies carrier screening
Up to 71% of people carry at least one pathogenic variant that could contribute to development of a…
Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study
Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results from its Phase 3 registrational clinical trial (the RECONNECT Study)…
Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study
Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results…
Saol Therapeutics hit with CRL for rare genetic disease
The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s…
Insights into clinical features and genetic variants of Cornelia de Lange syndrome in China
Cornelia de Lange syndrome (CdLS) is a rare genetic disorder with symptoms, including facial anomalies (such as fused…
Gene-Therapy Treatment Of Infant With Rare Genetic Condition Successful In World First » TwistedSifter
Pexels When baby KJ was born with a rare disease, which could cause a potentially fatal amount of…
Researchers find CFI deficiency alarmingly high in old order Amish
Researchers from the Children’s Hospital of Philadelphia (CHOP) and the Clinic for Special Children found that complement factor…
Anderson family battling 3-year-old’s rare gene disorder | Local News
ANDERSON — An Anderson family is battling their 3-year-old son’s rare brain-development condition. It’s believed that Beau Hanson…