Please be aware these summaries are produced voluntarily by Biomedical Science students and are their interpretations of the information and findings. This information is reviewed by our Research Analyst Kristina Backlund MMSc. The National Lottery Community Fund and Ionis Pharmaceuticals, Inc have contributed to the funding of the Research Analyst role.
Alex TLC assumes no responsibility or liability for any errors or omissions in the content of these summaries. The information contained in the research summaries is provided on an “as is” basis with no guarantees of completeness, accuracy, usefulness, or timeliness. If you are unsure of any of the details within the summaries, please refer to the actual articles or contact info@alextlc.org
We have developed a database of research summaries that include leukodystrophy articles, clinical trials and pharmaceutical press releases to allow our community to easily read about relevant recent research developments.
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Alexander disease (AxD) https://ir.ionis.com/news-releases/news-release-details/zanvastrotm-zilganersen-approved-fda-first-and-only-disease The U.S. Food and Drug Administration (FDA) has approved ZANVASTRO™ (zilganersen) for people living with Alexander diseasein the United States, making it the first approved treatment for this rare leukodystrophy. ZANVASTRO was safe and well tolerated in trials with statistically significant stabilisations of speed of walking and improved gross motor function […]
Alexander disease (AxD) https://www.sciencedirect.com/science/article/pii/S1096719226004725 Alexander disease (AxD) is a rare leukodystrophy, where Type 1 AxD usually begins in infancy or early childhood and is caused by pathogenic variants, in the GFAP gene, which provides instructions for making glial fibrillary acidic protein (GFAP) in astrocytes. Pathogenic variants are changes in the DNA that have been confirmed […]
Alexander disease (AxD) https://ir.ionis.com/news-releases/news-release-details/ionis-announces-license-agreement-recordati-zilganersen Ionis have entered into a license agreement with Recordati to commercialise zilganersen for Alexander disease for all countries outside of the United States. This is a significant step towards providing access to a disease modifying treatment for Alexander disease patients.
Alexander Disease (AxD) https://onlinelibrary.wiley.com/doi/10.1002/acn3.70305 Alexander disease (AxD) is a rare genetic disorder that affects the brain and spinal cord. It is caused by changes in the GFAP gene, which leads to an abnormal build-up of a protein called glial fibrillary acidic protein (GFAP) in brain cells known as astrocytes. These cells normally support and protect […]
Alexander Disease (AxD) https://ir.ionis.com/news-releases/news-release-details/ionis-announces-zilganersen-new-drug-application-alexander The U.S. Food and Drug Administration (FDA) has accepted Ioni’s New Drug Application (NDA) for zilganersen, the investigational treatment for Alexander Disease for Priority Review. The acceptance of the NDA is the critical first step in the FDA review process. The FDA will now begin a thorough review of the application. Priority Review […]
Alexander Disease https://www.sciencedirect.com/science/article/pii/S109671922500681X?via%3Dihub#ab0005 Alexander disease is a rare, progressive neurological condition caused by changes, known as variants, in the GFAP gene. This gene provides instructions for making glial fibrillary acidic protein (GFAP), a key structural protein in astrocytes, which are support cells in the brain. When GFAP is altered, it can form abnormal clumps called […]
Alexander Disease https://alextlc.org/wp-content/uploads/2026/01/Community-Statement-Ionis-Opens-EAP-in-the-US-for-Zilganersen-12.19.25.pdf Ionis has established an Expanded Access Program (EAP) in the United States for people with Alexander Disease to access zilganersen. An Expanded Access Program is a pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product for treatment outside of clinical […]
Alexander disease (AxD) https://onlinelibrary.wiley.com/doi/10.1002/acn3.70209 Alexander Disease (AxD) is a rare, genetic neurological disorder caused by mutations in the GFAP gene. It primarily affects astrocytes and leads to symptoms such as developmental delays, seizures, and a severe problem known as failure to thrive, where affected children struggle to gain weight and maintain muscle and fat. This […]
Alexander disease (AxD) https://ir.ionis.com/news-releases/news-release-details/ionis-announces-positive-topline-results-pivotal-study Ionis have shared results from their study of Zilganersen in children and adults with Alexander Disease. Zilganersen demonstrated statistically significant and clinically meaningful stabilisation on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test (10MWT) compared to the control group with favourable safety and tolerability. It also showed […]
Alexander Disease https://pn.bmj.com/content/early/2025/05/10/pn-2024-004490.long Alexander disease (AxD) is a rare genetic disorder caused by mutations in the GFAP gene. It is traditionally classified into infantile, juvenile and adult forms. While early-onset cases are often severe, adult-onset AxD is very diverse in how it presents and can be mistaken for other conditions such as multiple sclerosis. In […]
