On the very day Noa was diagnosed, an experimental gene therapy and clinical trial were posted to treat Canavan disease. Noa was only the third patient in the world to be dosed with the gene therapy, ...
The U.S. Embassy in Bangkok is warning Americans of potential "violent retaliatory attacks" after a group of Uyghurs were ...
The Novato-based maker of biopharmaceutical treatments for rare diseases plans to release this year a new gene therapy for ...
According to an affidavit obtained by People, Arakawa “showed obvious signs of death, body decomposition, bloating An influencer's trip to Disneyland quickly turned from magical to mortifying. The US ...
Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...
New research has looked into a comprehensive and structured approach to genetic testing, matching children and young people ...
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the ...
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...
Iovance Biotherapeutics, Inc. sees mixed TIL therapy success with Amtagvi in PD-1-refractory melanoma. Click here to find out ...
A comprehensive study of effective responses as well as resistant responses to a kidney cancer anti-PD-1 therapy found ...
A 24-year-old woman from York has become one of the first patients in the world to take part in a pioneering research study ...