Can ultrasound treat Duchenne? Sonothera raises millions to find out
For the last year, a small California startup has been making extraordinary claims about the ability of its…
MEP calls for Ireland to increase testing done on newborn babies for rare conditions – The Journal
MEP calls for Ireland to increase testing done on newborn babies for rare conditions The JournalCork MEP Billy Kelleher…
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…
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…
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”…
Coverage of Sarepta Duchenne drug may be halted by N.Y. state Medicaid
In a setback for Sarepta Therapeutics, a New York panel has voted unanimously to recommend the state Medicaid…
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…
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…