Sentences with word «adrenoleukodystrophy»

Led by Florian Eichler, MD, from Harvard Medical School and Massachusetts General Hospital, the study tested the first successful gene therapy treatment for cerebral adrenoleukodystrophy (ALD), a fatal degenerative brain disease that most severely affects boys.

The 1992 tearjerker Lorenzo's Oil told the true story of one family's struggle to save their son from X-linked adrenoleukodystrophy (ALD), a deadly degenerative brain disease.

Working under the deadline of a fatal illness, the Odones created a novel treatment for adrenoleukodystrophy, which disrupts fat metabolism, primarily in boys.

As an example of the complex problems that could arise, the report describes the hypothetical case of a man with a mild form of adrenoleukodystrophy who wants the fact kept secret.

Scientists have created a mutant fruit fly that suffers from symptoms resembling adrenoleukodystrophy (ALD), a rare, fatal hereditary brain disease made famous by the 1993 movie Lorenzo's Oil.

Now, as they get ready to celebrate the first birthday of their healthy son Gus, and the arrival of his sibling conceived the same way, they know that they've stopped adrenoleukodystrophy, or ALD, from traveling further down their family line.

In a groundbreaking new study, doctors were able to use gene therapy to halt the progress of a fatal neurodegenerative brain disease called adrenoleukodystrophy, or ALD.

Untreated cerebral adrenoleukodystrophy (ALD) leads to severe disability or death approximately 2 years after its onset.

• Combining stem cells with gene therapy, an international collaboration announced the success of a pilot study to treat X-linked adrenoleukodystrophy (ALD), a fatal brain disease caused by a mutation of the gene coding for the ALD protein.

X-linked adrenoleukodystrophy (X-ALD) is the most prevalent peroxisomal disorder.

Problems with myelin have also been linked to fibromyalgia, adrenoleukodystrophy (ALD), Krabbe disease, Charcot - Marie - Tooth disease (CMT), Guillain - Barré Syndrome, small fiber neuropathy and chronic inflammatory demyelinating polyneuropathy.

She'd never heard of the disease — adrenoleukodystrophy, or ALD — and she soon felt devastated and overwhelmed.

It is based on the real - life experiences of Michaela and Augusto Odone, whose son Lorenzo was diagnosed as having adrenoleukodystrophy (ALD) in 1984.

In the film it is portrayed as having stopped the progress of the degenerative disease adrenoleukodystrophy (ALD) in a boy called Lorenzo Odone.

The New York Times reports that a team led by Boston Children's Chief Scientific Officer David Williams, MD, have used gene therapy to stave off a fatal degenerative brain disease - adrenoleukodystrophy (ALD)- an achievement that some experts had thought impossible.

Washington, D.C. — April 18, 2018 — The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation's leading academic health centers, today awarded its most prestigious honor to a Massachusetts General Hospital research team for its discovery of the first successful gene therapy treatment for a fatal brain disease, cerebral adrenoleukodystrophy (ALD).

Previously the long - term benefits of hematopoietic stem - cell transplantation in cerebral adrenoleukodystrophy (ALD) are thought to be mediated by donor - derived replacement of myeloid derived cells, possibly including microglial cells.

In fact, erucic acid is helpful in the treatment of the wasting disease adrenoleukodystrophy and was the magic ingredient in Lorenzo's oil.