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 ...

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 ...

Among members of the Richardson family who carry a mutation in the PSEN1 gene, the average age when symptoms start is 39.

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

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

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

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

The pivotal trial for Neurogene's Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that ...