Becoming the first treatment for rare genetic disorder Prader-Willi syndrome, breakthrough therapy drug DCCR (diazoxide choline) gained U.S. FDA approval as Vykat XR the evening before its March 27 ...

The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...

The U.S. Food and Drug Administration approved Soleno Therapeutics' drug to treat a rare genetic disorder on Wednesday, ...

She announced Arakawa’s cause of death was hantavirus — a rare illness contracted via contact with rodents like rats and mice. Meanwhile, Hackman’s death was tied to heart disease with ...

Today, the Government of Canada and the territory of Yukon signed the National Strategy for Drugs for Rare Disease (DRD) agreement to invest over $8.5 million over three years to improve access to new ...

A primary growth driver for the Mepsevii market is the rising prevalence of mucopolysaccharidosis (MPS) disorders. These genetic conditions ... facilitating access to rare disease treatments.

These pages give an overview of what genes are and how they are involved in the different diseases that cause dementia. You will also find information about genetic testing for dementia. Genes ...

Latest research led from Manchester could revolutionise the diagnosis of Perrault syndrome, a rare genetic condition that results in hearing loss. In women it also leads to early menopause or ...

Childs Cure co-founders Jainu and Shruti Jogani founded the organization to address the genetic causes of rare diseases that are often under-researched, leaving people with few to no treatment ...

These databases have become invaluable resources for clinicians, researchers, and pharmaceutical companies striving to develop innovative treatments for genetic conditions. Global Rare Disease ...

"We are incredibly proud of our partnership with Jupiter and look forward to the work they are doing to bring life-changing solutions for rare ... Disease, Parkinson’s Disease ...