1st March 2021
The first patient has been treated in a clinical trial of a gene therapy for Tay-Sachs and Sandhoff Disease
GM2 gangliosidosis
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1st March 2021
GM2 gangliosidosis
1st March 2021
Cerebrotendinous Xanthomatosis (CTX)
1st March 2021
Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL)
1st March 2021
Adrenomyeloneuropathy (AMN)
1st March 2021
Adrenoleukodystrophy (ALD)
1st March 2021
Adrenoleukodystrophy (ALD)
1st March 2021
Adrenoleukodystrophy (ALD)
1st March 2021
Research summary of recent leukodystrophy research and clinical trials, includes article summaries and direct links to websites and articles.