For those people struggling with GNE myopathy, there is someone who wants to hear from you. Whether you’re a patient or a loved one of someone with this rare disease, a group of people wants to hear all about your story—right down to the nitty-grittiest detail. And it’s not for their gain necessarily, you could benefit too, according to an online article on TREAT-NMD.
GNE myopathy is a very rare disease that causes a person to progressively lose muscle over their lifetime. You can read more about it here.
It’s caused by a mutation in the GNE gene, and it affects sialic acid production. GNE myopathy causes muscle weakness (starting around age 20 or 30) in the lower and upper parts of the body. Currently, there are no approved treatments for GNE myopathy. And that’s where you come in…
Your story matters. Your feedback counts. The answer to unlocking the cure to GNE myopathy may be hidden in the smallest detail of your experiences.
Scientists and researchers want to know about you and your condition, to get a better picture of this disease and how it affects your life. To gather this important information, they have created online registries for you to record your health history in a secure online data registry.
The registries have a number of benefits for those who participate.
You may be contacted if you fit the criteria for an upcoming study or trial.
You can choose to be notified of new treatment developments.
Your participation is voluntary and can be withdrawn at any time.
Although the database is operated in Japan, the goal is to gather health information worldwide to offer the most diverse collection.
The registry is an international resource for people living with GNE myopathy, and researchers seeking the next breakthrough for GNE myopathy. Where will the next development come from? It could be you.