How tailored genetic therapy offers hope for rare disorders
Seven-month-old Kyle “KJ” Muldoon Jr was born with a life-threatening rare genetic disorder. This disorder was caused by…
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…
Fundraiser for local toddler battling rare genetic disorder
BRIGHT, Ind. (WKRC) – At just one year old, Van Foster has already taught his parents, Kristen and…
Prenatal diagnosis of over 1,700 foetuses finds one in four suffering from genetic anomalies | Ahmedabad News
A 14-year-long study on prenatal diagnosis (PND) of rare genetic disorders, conducted by a leading Ahmedabad-based institute and…
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…
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…
Is Autism a Genetic Disorder? Expert Shares Fact From Fiction
Autism is a topic many families think about, especially when a child shows signs of developmental differences. People…