Gene therapy makes major stride in ‘Lorenzo’s Oil’ disease

In the third gene-therapy success of recent weeks, French researchers have arrested the progression of the rare and fatal degenerative disorder adrenoleukodystrophy, which was at the heart of the popular movie “Lorenzo’s Oil.” The disease has stabilized in two boys who were 7 years old . . . → Read More: Not directly FX related but it has potential.

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Clinical Tests Begin on Medication to Correct Fragile X Defect

NIH-supported scientists at Seaside Therapeutics in Cambridge, Mass., are beginning a clinical trial of a potential medication designed to correct a central neurochemical defect underlying Fragile X syndrome, the most common inherited cause of intellectual disability. There has . . . → Read More: Clinical Tests Begin on Medication to Correct Fragile X Defect