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…
11-Month-Old Girl Diagnosed with Rare Genetic Disorder That Affects Fewer Than 100 People: ‘Breaks My Heart,’ Mom Says
NEED TO KNOW Kayleigh and Ryan Dunn’s 11-month-old daughter daughter, Lorelei, was diagnosed with the rare genetic disorder…
11-Month-Old Girl Diagnosed with Rare Genetic Disorder That Affects Fewer Than 100 People: ‘Breaks My Heart,’ Mom Says
Kayleigh and Ryan Dunn with their daughter, LoreleiCredit: GoFundMe NEED TO KNOW Kayleigh and Ryan Dunn’s 11-month-old daughter…
U.S. Cell And Gene Therapy Clinical Trials Market Trends
Dublin, Oct. 03, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell And Gene Therapy Clinical Trials Market Size, Share…
U.S. Cell And Gene Therapy Clinical Trials Market Trends
Dublin, Oct. 03, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell And Gene Therapy Clinical Trials Market Size, Share…
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)…
Genetic testing pilot aims to catch a silent killer
FH Education Message
A potentially life-saving pilot program is launching in Bloomington, Illinois to…
US FDA declines to approve higher dose of Biogen’s genetic disorder drug
(Reuters) -The U.S. Food and Drug Administration has declined to approve a higher-dose version of Biogen’s drug for…
The genetic answer to rare diseases
The standard treatment is a liver transplant, but this entails a high risk of complications in infants. Thanks…
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…
She has a rare genetic metabolic disorder but fought all odds to live and is now 17
SINGAPORE – When people hear the word “MMA”, they likely think of mixed martial arts, a full-contact combat…
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…