👉 👉 FDA has granted accelerated approval to the novel gene therapy elivaldogene autotemcel for the treatment of cerebral adrenoleukodystrophy, a rare, progressive, neurodegenerative disease mostly affecting young boys. FDAapproves GeneTherapy
September 19, 2022
The US Food and Drug Administration has granted accelerated approval to the novel gene therapy elivaldogene autotemcel for the treatment of cerebral adrenoleukodystrophy . The therapy"is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age" with early, active CALD, the manufacturer noted in a press release.
CALD is a rare, progressive, neurodegenerative disease that mostly affects young boys and causes irreversible neurologic decline, including major functional disabilities such as loss of communication, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement.
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