Genetix of Cambridge invests $35m in 2 new gene therapies Aug 2, 2010
Left untreated, both diseases the blood disorder beta-thalassemia major and the brain-wasting disease Adrenoleukodystrophy, or ALD are fatal. And existing treatments can be as dangerous as the diseases. (Boston Globe)
Okla. couple's 3 children struck by fatal disease May 24, 2010
All three boys have ALD, or adrenoleukodystrophy, a rare, inherited disease that leads to brain damage and death. Eleven-year-old Anthony Middleton, once a football-throwing, video-game playing ball of energy, was diagnosed in January. (Alva Review Courier, OK)
Genetix raises $35m for gene therapy work Mar 15, 2010
The Cambridge-based company attracted the fresh capital after a study in France showed that one of the firm s gene therapies blocked the progression of a debilitating brain disorder called adrenoleukodystrophy, or ALD, in two children. This is the disease that was featured in the acclaimed 1992 film Lorenzo s Oil, the true story of a husband and wife who searched for a cure for their son with the crippling illness. (Boston Globe)
Gene Target Beats Oil Remedy Mar 13, 2010
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. Unfortunately, over the ensuing years, the oil of the film s title, a dietary supplement, has not panned out as the cure many people hoped it would be. (Scientific American)
Lovastatin in X-Linked Adrenoleukodystrophy Jan 21, 2010
To the Editor: As reported previously in the Journal, lovastatin lowers levels of very-long-chain fatty acids in plasma in patients with X-linked adrenoleukodystrophy (X-ALD). 1 Further studies did not reproduce this finding with the use of simvastatin in patients or with the use of lovastatin in X-ALD knockout mice. (New England Journal of Medicine)
'Lorenzo's Oil' Breakthrough: Newfound Mechanism Could Prevent or Treat Deadly Peroxisome Diseases Jan 10, 2010
One peroxisome disorder is adrenoleukodystrophy, or ALD, the disease a boy named Lorenzo Odone suffered from. His parents' fight for a cure was made into the movie. (Science Daily)
Nature Medicine Jan 10, 2010
Two boys with X-linked adrenoleukodystrophy, the disease highlighted in the movie "Lorenzo's Oil" received gene therapy, and their outcome was similar to that achieved by conventional HSC-transplantation treatment. What do the data mean. (Nature News Service)
Gene Therapy for Fatal Brain Disorder 'Just the Beginning' Jan 10, 2010
Pioneering work may offer hope for patients with adrenoleukodystrophy ... THURSDAY, Nov. 5 (HealthDay News) -- The reported success of gene therapy in treating two children with adrenoleukodystrophy (ALD) brings hope to patients with the potentially crippling and fatal brain disorder and their families, says a nonprofit group that supports ALD research. (MEDLINEplus)
