He seemed drunk without taking a sip of alcohol. It took years to get the rare illness diagnosed
For nearly 10 years, Eric Poulin has been having episodes where he seems drunk — without taking even…
Passengers begin leaving hantavirus ship at Tenerife – POLITICO
Spanish Health Minister Mónica García said the disembarkation was “proceeding normally” and that all passengers on board the…
Intellia Therapeutics moves HAE treatment closer to approval
Intellia Therapeutics said Monday that a single dose of its gene editing treatment dramatically reduced swelling attacks in…
FDA unveils new pathway for rare disease treatments
The Trump administration on Monday released detailed guidance for approving the first bespoke medicines crafted to treat patients’ individual…
FDA rejects Regenxbio’s MPS II rare-disease gene therapy
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Alternate proteins from the same gene contribute differently to health and rare disease | MIT News
Ly shows that one function this serves is to send versions of a protein to different parts of…
Man dies in first known fatality from tick-borne meat allergy
NEWYou can now listen to Fox News articles! A New Jersey man’s death is the first known to…
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”…
Experimental treatment restores sight to man with genetic eye disorder in Italy
By Euronews Published on 30/07/2025 – 13:02 GMT+2 ADVERTISEMENT In a world first, a man suffering from vision loss…
FDA clears way for Sarepta Therapeutics to use Duchenne drug in some patients
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Sarepta Therapeutics’ Elevidys faces ‘arduous’ path back to market
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Sarepta will stop shipping Elevidys, Duchenne gene therapy
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…