Accumulation of somatic mutations drives vascular damage in people with progeria
Hutchinson-Gilford progeria syndrome (HGPS) is a genetic disorder that causes remarkable premature aging. Most patients die during their…
Gene therapy successfully treats deadly childhood liver disease in mice
A new gene therapy has been used to successfully treat a deadly childhood liver disease using mice that…
Neurocrine to Buy Soleno, Nabbing Drug for Relentless Hunger Disorder
The deal is the latest in a busy year for dealmaking in the biotech industry. – eric piermont/Agence…
Toxic RNA drives progressive heart damage in myotonic dystrophy
Myotonic dystrophy type 1 (DM1) is the most common cause of adult-onset muscular dystrophy, a genetic disorder that…
Updated newborn screening guidelines aim to improve cystic fibrosis detection
An updated report on cystic fibrosis (CF) from Ann & Robert H. Lurie Children’s Hospital of Chicago, in…
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…
Genetic testing pilot aims to catch a silent killer
FH Education Message
A potentially life-saving pilot program is launching in Bloomington, Illinois to…
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…