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1st August 2021

GM1 gangliosidosis Clinical Gene Therapy Trial

GM1 gangliosidosis

1st August 2021

Clinical Stage Gene Therapy Program for the treatment of GAN

Giant Axonal Neuropathy (GAN)

1st August 2021

Clinical trial to validate new gene therapy for treatment of Canavan Disease

Canavan Disease

1st August 2021

Natural History of Canavan Disease; new cases and comparison with patients from literature

Canavan Disease

1st August 2021

Clinical study of medicine to treat Alexander Disease

Alexander Disease

1st August 2021

Preclinical evidence of drug to treat AMN in mice models

Adrenomyeloneuropathy (AMN)

1st August 2021

Drug for the effective treatment of X-ALD

Adrenoleukodystrophy (ALD)

1st August 2021

Gene rescue potential therapeutic strategy for ALD treatment

Adrenoleukodystrophy (ALD)

1st August 2021

Preclinical programs demonstrate potential gene therapies for the treatment of APBD and GM2

GM2 gangliosidosis

1st August 2021

Research Summary: August 2021

Research summary of recent leukodystrophy research and clinical trials, includes article summaries and direct links to websites and articles.

1st July 2021

Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease

Pelizaeus-Merzbacher Disease (PMD)

1st July 2021

Forge Biologics Receives FDA Fast Track, Orphan Drug and Rare Paediatric Disease Designations

Krabbe Disease

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