Researchers at Memorial Sloan Kettering Cancer Center (MSK) have developed some of the first robust laboratory models of a ...

Understanding how cells turn genes on and off is one of biology's most enduring mysteries. Now, a new technology developed by ...

Recently approved by the European Medicines Agency, RESTORE will evaluate iadademstat’s ability to raise fetal hemoglobin (HbF) in adult patients ...

Recently approved by the European Medicines Agency, RESTORE will evaluate iadademstat's ability to raise fetal hemoglobin (HbF) in adult patients with SCDIadademstat has demonstrated a favorable safet ...

A Northwestern Medicine-led study has identified mutations in the gene CEP76 as a new cause of ciliopathies, shedding light on a complex group of disorders that affect multiple body systems, according ...

Genetic testing is transforming healthcare through personalized medicine, enabling early diagnosis, targeted treatments, and a future of more precise, patient-centered care.

A company’s plan to edit the genomes of human embryos worries some researchers — but it might reflect the changing attitudes ...

Ion channel voltage-dependence alterations can shape intrinsic homeostatic plasticity, even if operating faster than other intrinsic regulatory processes—where timescale separation would suggest its ...

A new genomic method has enabled multiple people with rare conditions to receive diagnoses that were previously unattainable ...

Sarepta Therapeutics, Inc. , the leader in precision genetic medicine for rare diseases, today reported financial results for the third quarter of 2025 and the completion of ESSENCE, its global, Phase ...