GNE myopathy is an ultra-rare muscle disease with no cure so far. I am keen to understand scientific developments in GNE myopathy and related fields not just as a biomedical scientist, but also as a mother whose daughter lives with this progressive disease.
We are at a time when new scientific technologies are being created and developed at an incredible pace globally. Yet, many in the rare disease community do not have the scientific background to track and understand these scientific advancements.
We are at a time when new scientific technologies are being created and developed at an incredible pace globally. These bring hope of a treatment for rare genetic disorders that so far remained untreated. Yet, many in the rare disease community do not have the scientific background to track and understand these scientific advancements. Through this blog I hope to inform my readers about new scientific developments and outline those that may be promising avenues in the drug development process for GNE myopathy and related diseases. It is both a challenge and an opportunity to use my expertise to bring this information to my readers, with the final goal of finding a treatment for this, and related, muscular dystrophies. I hope for this to be an interactive dialogue on science and I encourage my readers to share their thoughts, suggestions or questions with me.