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1st February 2022

Passage Bio Announces 2022 Research and Clinical Development Goals to Advance Robust CNS Pipeline

GM1 gangliosidosis, Krabbe Disease & Metachromatic Leukodystrophy (MLD)

1st February 2022

Myrtelle Announces Successful Completion of Initial Stage of Phase 1/2 Clinical Trial of Proprietary Gene Therapy for Canavan Disease and Expands Treatment to Younger Patients

Canavan Disease

1st February 2022

Functional characterization of hiPSCs-derived glial cells and neurons from patients harboring a TREM2 loss of function mutation

Nasu-Hakola Disease (NHD)

1st February 2022

Parental Somatic Mosaicism Uncovers Inheritance of an Apparently De Novo GFAP Mutation

Alexander Disease

1st February 2022

Beneficial Effect of Phenytoin and Carbamazepine on GFAP Gene Expression and Mutant GFAP Folding in a Cellular Model of Alexander’s Disease

Alexander Disease

1st February 2022

FDA cleared the Investigational New Drug application for SBT101, SwanBio Therapeutic’s gene therapy candidate for AMN

Adrenomyeloneuropathy (AMN)

1st February 2022

Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia: Review of Clinical Manifestations as Foundations for Therapeutic Development

Adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)/CSF1R related leukoencephalopathy

1st February 2022

Viking X-ALD Study Paused Over Conflicting Phase Interpretations

Adrenoleukodystrophy (ALD)

1st February 2022

Isoprostanoid Plasma Levels Are Relevant to Cerebral Adrenoleukodystrophy Disease

Adrenoleukodystrophy (ALD)

1st February 2022

Typical and atypical phenotype and neuroimaging of X-linked adrenoleukodystrophy in a Chinese cohort

Adrenoleukodystrophy (ALD)

1st February 2022

N-Acetylcysteine Reverses the Mitochondrial Dysfunction Induced by Very Long-Chain Fatty Acids in Murine Oligodendrocyte Model of Adrenoleukodystrophy

Adrenoleukodystrophy (ALD)

1st February 2022

AAV Vectors Advance the Frontiers of Gene Therapy

Gene Therapy

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