RTE Online8 天
Wheelchair user without power for 11 days says she feels 'forgotten'
A wheelchair user in Athenry, Co Galway, said she feels forgotten about on her 11th day without electricity since Storm Éowyn ...
MedCity News6 天
Denali’s Data in Rare Enzyme Deficiency Keep It on Pace to Seek Speedy FDA Approval
Regenxbio has a commercialization partner for its Hunter syndrome gene therapy as well as RGX-111, a gene therapy for the enzyme deficiency known as Hurler syndrome. Per deal terms announced in ...
来自MSN1 个月
Christmas joy for boy after stem cell transplant
A five year-old boy with a life-limiting metabolic disorder is celebrating Christmas, after a vital stem cell transplant. In 2021, Gunner from Highley, Shropshire, was diagnosed with ...
The Business Journals28 天
Rockville’s RegenxBio strikes potential $810M deal with Japan’s Nippon Shinyaku
Ltd. will develop and commercialize its one-time gene therapies for Hunter syndrome and Hurler syndrome, both affecting children. Both are rare genetic disorders called mucopolysaccharidoses ...
FierceBiotech29 天
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
That partner, Japan’s Nippon Shinyaku, is handing over $110 million upfront to lead on the commercialization of two gene therapies—RGX-121 for Hunter syndrome and RGX-111 for Hurler syndrome.