Skysona is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy.

The agency is also evaluating if there's a need for further regulatory action based on reports of hematologic malignancies from clinical trials of the therapy.

The FDA last week announced that it is looking into the safety of bluebird bio ’s gene therapy Skysona (elivaldogene ...

The U.S. Food and Drug Administration is weighing the need for regulatory action on bluebird bio's gene therapy for a rare ...

Share on Facebook. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA said on Wednesday that it is investigating reports ...

Thus, CD36 is uniquely positioned at the crossroads of VLCFA uptake and regulation of the inflammatory response and may impact ALD pathology through either of these mechanisms. More work is ...

1. In this prospective cohort study, in boys with diagnosed cerebral adrenoleukodystrophy, it was found that lentiviral gene therapy containing ABCD1 complementary ...

While investigating a rare developmental disorder, researchers ended up discovering a spectrum of conditions that are all linked to one gene. When you purchase through links on our site ...

Scientists recreate mouse from gene older than animal life. ScienceDaily . Retrieved December 13, 2024 from www.sciencedaily.com / releases / 2024 / 11 / 241118125716.htm ...