Study reveals genetic factors influencing SYNGAP1 encephalopathy disease severity
SYNGAP1 encephalopathy is a rare genetic disorder for which there is no treatment, causing epilepsy, intellectual disability, psychomotor…
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…
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…
Gut microbial imbalance linked to chronic sickle cell disease pain
A research collaboration involving scientists and students at The University of Texas at Dallas has found a clear…
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…