Leukodystrophy Awareness Month (30 September 2025)
- Release Tx
- Sep 30
- 2 min read
The white matter of the brain was once disregarded by scientists as passive tissue and secondary to grey matter, where neurons reside. Leukodystrophies, a broad spectrum of rare genetic disorders affecting the white matter of the brain, have played a pivotal role in helping us understand its importance. As September and Leukodystrophy Awareness Month draw to a close, we are committed to ensuring that awareness continues into the months ahead and that we honour this hard-won knowledge by working towards greater care, continued research, and lasting support for those who live with these conditions.
Leukodystrophies are a broad spectrum of over 50 rare, inherited disorders of the central nervous system (CNS), characterised by the loss or impaired formation of myelin, a protective sheath that wraps around and protects each nerve cell.[1]
Myelin is found in the brain’s white matter, which makes up about half of the human brain, and is found in the deeper, subcortical tissues.[2] In fact, myelin gives the white matter its colour (thus "leuko", meaning "white", and "dystrophy", meaning progressive loss or weakness). An insulating material rich in lipids, myelin protects nerve cells and ensures the proper transmission of electrical impulses throughout the nervous system. When it breaks down or its production becomes impaired, neurotransmission of electrical impulses in the brain, spinal cord and nerves slows down or stops, leading to significant disruption in the normal functioning of the nervous system.
Whether they are caused by loss of the myelin sheath after its formation (demyelination) or incomplete formation of myelin (hypomyelination), leukodystrophies are typically caused by an inherited, defective gene involved in the development of myelin.[3] Each form of leukodystrophy is rare, but studies place the overall prevalence of leukodystrophies at 1 per 7,600 births, with most of these disorders associated with severe progressive losses of motor and cognitive ability, and premature death.[4] Crucially, most leukodystrophies affect young children.
As Release Therapeutics, we are proud to recognise September as Leukodystrophy Awareness Month, a time designated by advocacy groups to shine a light on these devastating and often misunderstood rare disorders. Raising awareness is critical because leukodystrophies often go undiagnosed or misdiagnosed, delaying time-sensitive intervention and affecting individuals and families in ways that most cannot imagine.
By spreading the word, we aim to bring attention to the need for better diagnostics, research, and, most importantly, new treatments. Right now, options are limited, and we’re committed to pushing forward in developing therapies that could slow, stop, or even reverse the damage caused by leukodystrophies. While the challenges are great, so too is the potential for progress.
Stay tuned for updates on how our implantable cell macroencapsulation platform is changing the outlook for rare CNS disorders.
The Release Tx Team