X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...
Quentin M. Anstee, B.Sc., M.B.B.S., Ph.D., M.R.C.P.; Ann K. Daly, B.Sc., Ph.D.; Christopher P. Day, B.A.,M.B., B.Chir.,M.A. (Cambridge), Ph.D.,M.D., F.R.C.P., F.Med ...
US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The EMA's ...
Inspired by their 9-month old son William, who was diagnosed with ALD after a newborn screening, the Hall family seeks to jumpstart research into the rare disease, in partnership with The Dallas ...
Allogeneic hematopoietic stem cell transplantation (HCT) has been shown to have disease-modifying effects in the demyelinating cerebral form of X-linked adrenoleukodystrophy (X-ALD), but finding a ...
GlobalData on MSN3mon
Bluebird bio’s Skysona led to seven cases of blood cancer in gene therapy trialsThe patients received the one-time autologous hematopoietic stem cell-based gene therapy as a treatment for early cerebral ...
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