This Sunday marks World ALS Day with emphasis on new genetic treatments that have stabilized or restored motor functions in some patients.
Amyotrophic lateral sclerosis affects seven out of every hundred thousand people worldwide and remains without a cure. Yet advances in therapies targeting molecular causes have shown positive results in clinical trials.
Doctor Brigitte van Zundert from Universidad Andrés Bello noted that treatments based on antisense oligonucleotides have allowed patients with SOD1 gene mutation to stabilize or improve motor and respiratory capacity after one year of application. Some even regained the ability to walk.
Her research identifies inorganic polyphosphate released by astrocytes as a cause of motoneuron death. This finding points to possible biomarkers for earlier diagnosis.
The ELA Chile corporation supports patients and families while gaps persist in access to innovative treatments compared to developed countries.
