Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are ...

A new gene therapy delivery device could let hospital pharmacies make personalized nanomedicines to order. This democratized ...

Using an AI-driven drug discovery process, the drug known as vorinostat was identified as a promising treatment for Rett syndrome.

Researchers at the Gray Faculty of Medical and Health Sciences at Tel Aviv University have developed a model that accurately ...

Ajay Sood emphasized the need for affordable cell and gene therapies in India, suggesting a national mission to drive down ...

A recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of ...

We look at the continued turmoil within the FDA, including the forced dismissal of top gene therapy regulator Nicole Verdun, ...

A researcher says an experimental gene therapy for a rare inherited disorder is saving almost as much money for the treatment ...

HealthDay News — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront. The deal gives Lilly a potential new treatment for heart disease, The Wall ...

A delay, as opposed to a firm stagnation, then, but one unfolding over several years. This applies not only to SMEs, but to ...