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Read More Here: https://giving.massgeneral.org/stories/ald-gene-therapy-and-the-gift-of-a-future?cid=dev4126f&utm_medium=social&utm_source=facebook&utm_campaign=mgh-development&fbclid=IwAR1-kQ8IGakwsBwl_CigazZyxZwu1NZMSCFd3UU7e7CqVsCPWUy_sdvAuRw

- Frances Broussard Denenburg

ALD: Gene Therapy and the Gift of a Future

An experimental gene therapy developed by Mass General’s Florian Eichler, MD, is giving hope to patients and families impacted by ALD — a fatal brain disease affecting boys under the age of 12.

FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy

If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death

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