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…
Montana Girl Diagnosed With Rare ReNU Syndrome | News
Kelli Stanley remembers the phone call as nothing short of an answered prayer. For nearly a decade, her…
CRISPR gene editing reshaping medicine, offering hope for genetic diseases
CRISPR technology is transforming modern medicine by enabling precise DNA editing to treat genetic disorders, including sickle cell…
Research, support and early diagnosis crucial for rare chromosome disorders | News
Rare Chromosome Awareness Day, celebrated in June, is an opportunity to increase public knowledge about rare chromosome and…
First Awareness, Then Action: The Life-Saving Power Of Cancer Genetic Screening | Marketplace
Photo Caption: Dr. Shoshana Ungerleider, MD—San Francisco internist and jscreen.org board member—shares how hereditary testing saves lives during…
Screening for genetic mutations in couples before they conceive
Couples can unknowingly be carriers for genetic conditions, even though there are tests available to reveal recessive traits…
Sickle Cell Medication Cost Soars – NGO Plea to Nigeria
A Non-governmental Organization, Living Hope Save Cell Foundation, has lamented the high cost of medication for the patients…