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, ...
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
The Novato-based maker of biopharmaceutical treatments for rare diseases plans to release this year a new gene therapy for ...
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the ...
ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...
Columnist Jennifer Lynne assesses the effects of Pfizer's decision to halt production of its hemophilia B gene therapy, Beqvez.
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal ...
Krystal Biotech said the European Medicines Agency's Committee for Medicinal Products for Human Use has recommended approval of its Vyjuvek topical gene therapy for certain patients with the rare ...
A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...
My brothers and I compare our genetic analyses and what diseases we are genetically at risk for and what conditions we ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback