What a shame .

This is a quote from the paper linked below:

Trehalose reduces aggregate formation and delays pathology in a transgenic mouse model of oculopharyngeal muscular dystrophy

As one can identify almost all cases at risk, this disease is amenable to pre-symptomatic treatment. If one can delay onset of a disease that typically presents around the age of 50 by 40 years, then one has effectively cured the disease. Trehalose would be particularly attractive for this strategy, given its safety and suitability for long-term use. However, the disease course in mice is very constricted when compared with the human situation, and we cannot guarantee that a treatment that is effective in mice will have a similar effect in humans. Also, such a treatment would need to be administered for decades in humans. Although trehalose is appealing in this context, as it appears to be safe at high doses in rodents and rabbits and is well tolerated at high doses in humans , further studies will be required to test that it is safe when administered over much longer periods.

For those who don’t know oculopharyngeal muscular dystrophy is a form of ataxia, and is the subject of this clinical trial:

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients (HOPEMD)

Tragically it may be yet another decade before we ataxians see this tested for other forms of ataxia, and worse yet it is being studied as a treatment for people for whom the damage has already been done and yet the real potential lies in helping those not yet symptomatic as the report above hypothesizes.

God Speed BioBlast!