Calico targets longevity pathway with rare disease win
FDA Breakthrough Therapy Designation for fosigotifator highlights a mechanism that extends well beyond an ultra-rare childhood disorder. Calico…
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…
Boy’s heart stops for 16 minutes after tube comes loose in Hong Kong hospital
A probe has been launched into a serious medical incident in which a six‑year‑old boy’s heart stopped for…
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…
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”…
Knowing patient’s genetic make-up may change the way medicines are prescribed
SINGAPORE – Not all medicines work the same way on every patient, and part of the answer lies…
Acadia Pharmaceuticals drug for rare genetic condition fails late-stage study
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
The genetic answer to rare diseases
The standard treatment is a liver transplant, but this entails a high risk of complications in infants. Thanks…
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…
World’s first gene therapy for p47 Chronic Granulomatous Disease developed by UCL and GOSH
image: ©UCL UCL and Great Ormond Street Hospital have developed the world’s first gene therapy for p47 Chronic…