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1st October 2021

Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 for Treatment of Krabbe Disease

Krabbe Disease

1st October 2021

IntraBio Reports Further Detail On Positive Data From IB1001 Multinational Clinical Trial For The Treatment of GM2 Gangliosidosis

GM2 Gangliosidosis

1st October 2021

Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis

GM2 Gangliosidosis

1st October 2021

Sio Gene Therapies Announces Dosing of First GM1 Gangliosidosis Early Infantile (Type I) Patient in Ongoing Phase 1/2 Study of AXO-AAV-GM1 Gene Therapy

GM1 Gangliosidosis

1st October 2021

Clinical-Stage AAV9 Gene Therapy Program, TSHA-120, for the Treatment of Giant Axonal Neuropathy, a Rare and Severe Neurodegenerative Disease

Giant Axonal Neuropathy (GAN)

1st October 2021

Riaan Research Initiative funds Cockayne syndrome gene replacement therapy research at UMass Chan Medical School

Cockayne Syndrome (CS)

1st October 2021

A case of CADASIL caused by NOTCH3 c.512_605delinsA heterozygous mutation

Cerebral Autosomal Dominant Arteriopathy with Subcortical infarcts and Leukoencephalopathy (CADASIL)

1st October 2021

X-linked adrenoleukodystrophy presenting as progressive ataxia and pure cerebellar involvement

Adrenoleukodystrophy (ALD)

1st October 2021

Translational and clinical pharmacology considerations in drug repurposing for X-linked adrenoleukodystrophy—A rare peroxisomal disorder

Adrenoleukodystrophy (ALD)

1st October 2021

Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy

Adrenoleukodystrophy (ALD)

1st October 2021

Vigil Neuroscience begin work to better understand ALSP in order to identify parameters which can be used to inform design of a clinical trial for a potential treatment.

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

1st October 2021

Analysis of brain MRI scans in pre-symptomatic X-ALD patients can be used to predict disease course and inform treatment options

Adrenoleukodystrophy (ALD)

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