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1st March 2022

Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis

GM2 gangliosidosis

1st March 2022

Sio Gene Therapies Announces Prioritization of Lead Gene Therapy Programs in GM1 and GM2 Gangliosidosis, Extension of Cash Runway, and CEO Transition

GM1 gangliosidosis & GM2 gangliosidosis

1st March 2022

Taysha Gene Therapies Reports Positive Clinical Efficacy and Safety Data for High Dose Cohort and Long-term Durability Data for TSHA-120 in Giant Axonal Neuropathy

Giant Axonal Neuropathy (GAN)

1st March 2022

Myrtelle Announces Positive Preliminary Clinical Data for Its Proprietary Gene Therapy in Canavan Disease

Canavan Disease

1st March 2022

Research Summary: March 2022

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

1st March 2022

Alexander disease evolution over time: data from an Italian cohort of paediatric-onset patients

Alexander Disease

1st March 2022

GFAP variant p. Tyr366Cys demonstrated widespread brain cavitation in neonatal Alexander disease

Alexander Disease

1st March 2022

Newborn Screening for X-Linked Adrenoleukodystrophy: Past, Present, and Future

Adrenoleukodystrophy (ALD)

1st February 2022

CWRU grants exclusive license to Ionis Pharmaceuticals to advance antisense therapy for Pelizaeus-Merzbacher disease

Pelizaeus-Merzbacher Disease (PMD)

1st February 2022

Orchard Therapeutics Announces Publication in The Lancet of Long-term Clinical Outcomes with Libmeldy for the Treatment of Children with Early-onset MLD

Metachromatic Leukodystrophy (MLD)

1st February 2022

Dementia protein changes discovered in terminal childhood disease

Krabbe Disease

1st February 2022

Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101, the First Bicistronic Gene Therapy in Clinical Development, Demonstrating Normalization of β-Hexosaminidase A Enzyme Activity in Patients With GM2 Gangliosidosis

GM2 gangliosidosis

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