Regenerative Therapy and Neuroprotection
We continue to advance regenerative approaches aimed at protecting vision and the nervous
system. One of our key efforts involves a naturally occurring neurotrophic factor called MANF, a
molecule that supports cell survival and recovery under stress. MANF is particularly relevant to
Wolfram syndrome because it activates pathways that help cells cope with endoplasmic
reticulum stress, a major contributor to vision loss and neurodegeneration.
The new findings from our optic atrophy study, described above, are directly informing this
work. If the earliest event in vision loss is a quiet weakening of synaptic connections rather than the death of retinal ganglion cells themselves, then regenerative and neuroprotective strategies
have a real window in which to act. With renewed support, our MANF program has regained
momentum, and we are steadily generating new data. We plan to share our progress at the
next Wolfram syndrome conference.
https://wolframsyndrome.co.uk/wp-content/uplo...Y-2026.pdf