News

The American Journal of Managed Care12h
5 Rare Disorders Named for Pioneering Female Scientists
Explore 5 rare genetic and neurological disorders named after pioneering women in medicine, highlighting their significant ...
Blood test offers faster, less invasive diagnosis for rare genetic diseases in children and infants
Researchers from the University of Melbourne and Murdoch Children's Research Institute (MCRI) have developed a blood test ...
AAP Newsroom on MSN1d
Rare genetic diseases rapidly detected under new test
Researchers have developed a new blood test capable of rapidly diagnosing rare genetic diseases in babies and children.
The BMJ4d
Personalised CRISPR therapy: Could it transform treatment of genetic disease?
An infant with a rare genetic condition made headlines last week after successfully receiving personalised CRISPR therapy. Jacqui Wise summarises what we know so far about the breakthrough US doctors ...
Fierce Biotech4d
Applied's rare disease drug suffers another phase 3 failure months after FDA rejection
Applied Therapeutics’ rare disease drug has failed another late-stage clinical trial, adding to the woes of a candidate that ...
Public Broadcasting Service (PBS)7d
Experimental gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions
Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...
Healio7d
Infant with rare disease receives world’s first personalized gene-editing therapy
An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...
FOX 10 Phoenix8d
Baby born with rare disorder thrives after getting personalized gene editing treatment
Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...
MedPage Today8d
In a First, CRISPR Used to Treat Infant With Ultra-Rare Disease
A tailored CRISPR base-editing therapy was given for the first time to an infant who was born with a rare genetic disease, with promising effects, researchers reported.