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Vanishing White Matter Disease (VWMD) https://www.calicolabs.com/press/calico-life-sciences-announces-fosigotifator-granted-breakthrough-therapy-designation-by-the-u-s-fda-for-the-treatment-of-vanishing-white-matter-disease Calico have been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for Fosigotifator, a potential treatment for Vanishing White Matter (VWM) disease. Breakthrough Therapy designation is intended to speed up the review of medicines that treat a serious or life-threatening condition and have shown preliminary […]

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.

Research summary of recent leukodystrophy research and clinical trials, includes article summaries and direct links to websites and articles. To access the summary: Research Summary – July 2026 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 […]

Krabbe Disease https://investors.polaryx.com/pr/polaryx-therapeutics-announces-closing-of-10-million-private-placement-equity-financing-to-support-launch-of-soteria-phase-2-basket-trial

L-2-hydroxyglutaric aciduria https://www.nature.com/articles/s41586-026-10564-x This study shows that L‑2‑hydroxyglutarate (L‑2‑HG), a molecule long believed to be only a toxic by‑product plays an important and normal signalling role inside cells. Under healthy conditions, L‑2‑HG is kept at very low levels because a mitochondrial enzyme called L2HGDH rapidly converts it back into another molecule, 2‑oxoglutarate. When this enzyme […]

GM1 gangliosidosis and GM2 gangliosidosis https://www.azafaros.com/news-media/azafaros-announces-publication-of-phase-2-rainbow-study-data-for-nizubaglustat-in-molecular-genetics-and-metabolism-journal/l275c14.

Krabbe Disease https://link.springer.com/article/10.1186/s13073-026-01620-2 Krabbe disease (KD) is a rare inherited neurological disorder caused by mutations in the GALC gene. These mutations prevent the body from producing enough GALC enzyme, leading to the accumulation of a toxic substance called psychosine. As psychosine builds up, it damages myelin, causing severe neurological decline, and loss of motor function. […]

GM2 gangliosidosis https://www.sciencedirect.com/science/article/pii/S2666379126001795 GM2 gangliosidosis, which includes Tay‑Sachs and Sandhoff disease, is a severe inherited condition where the brain cannot break down a fatty substance called GM2 ganglioside. As GM2 builds up, it damages nerve cells and leads to progressive neurological decline. There are currently no approved treatments that can stop or reverse the disease. […]

Adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)/CSF1R related leukoencephalopathy https://journals.viamedica.pl/neurologia_neurochirurgia_polska/article/view/111875/89457 CSF1R‑related leukoencephalopathy is a rare, inherited brain disease that causes progressive damage to the white matter. It usually begins in adulthood and leads to changes in behaviour, thinking, movement, and balance. The condition is caused by harmful variants in the CSF1R gene, which affect how […]

Adrenoleukodystrophy (ALD) https://pmc.ncbi.nlm.nih.gov/articles/PMC12893433 Adrenoleukodystrophy (ALD) is a rare inherited condition that affects the nervous system. In many boys and men with ALD, a serious form called cerebral adrenoleukodystrophy (CALD) can develop, causing rapid damage to the brain. Without treatment, CALD can lead to severe disability or loss of life within a few years. However, treatments […]

Research summary of recent leukodystrophy research and clinical trials, includes article summaries and direct links to websites and articles. To access the summary: Research Summary – June 2026 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 […]

GM1 gangliosidosis and GM2 gangliosidosis https://investors.polaryx.com/pr/polaryx-therapeutics-to-participate-in-national-taysachs-allied-diseases-association-annual-family-conference