China releases powerful DNA-screening AI tool for free to help fight rare diseases
For people navigating rare diseases, which are mostly genetic in origin, the search for answers is a gruelling…
Rare disease drugmakers seek exclusion from Medicare pricing plans
WASHINGTON — Biotech companies are lobbying the Trump administration to exclude treatments for rare diseases from programs that…
ARPA-H invests $160 million to make custom gene editing routine
ARPA-H, the U.S.’ “moonshot” agency for health research, announced Thursday that it will spend up to $160 million…
How one father built a biotech, Grace Science, to save his daughter
Highlights Matt Wilsey has spent a decade and $70 million trying to develop a cure for his daughter…
Long-Read DNA Test Could Replace 15 Existing Tests for Rare Diseases
Credit: Radboudumc Researchers in the Netherlands have developed a DNA test for rare diseases that can provide much…
A mother’s 20-year quest fuels a startling Duchenne breakthrough
A year after the worst day of her life, Debra Miller received a voicemail she couldn’t quite make…
Crowds could trigger Celine Dion during comeback, according to patient with her disease
NEWYou can now listen to Fox News articles! A former marathon runner who was diagnosed with the same…
Couple donates $11 million to Lurie Children’s Hospital
A couple with deep ties to the Chicago area is donating $11 million to Lurie Children’s Hospital in…
Pharma sounds alarm over EU plans to procure novel medicines – POLITICO
But the pharmaceutical industry argues that any changes should be made cautiously and with clear guidelines for everyone…
CRISPR used in landmark treatment to fix misspelling in baby’s genome
For the first time, scientists say they have reached into the genome of a severely ill child and…
GeneDx to Partner with Florida’s Sunshine Genetics Network, Launching Nation’s First State-Backed Genomic Newborn Screening Program
Florida Institute for Pediatric Rare Diseases and Representative Adam Anderson ushering in a new era of preventative care…
Only four people in Australia have been diagnosed with rare genetic disease CHOPS
Isla Steed is your fairly typical teenager, according to her mum. The 14-year-old is “a stubborn little person”…