Alexander's Way Research Fund is a nonprofit, charitable organization that promotes international collaboration among scientists, researchers, and patient advocacy groups, to speed up research, development, and delivery of treatments for children and adults affected by Bag3 myofibrillar myopathy (MFM6), and other neuromuscular diseases.
A child diagnosed with this rare disease finds that the medical community does not have any treatment or cure. This reality is indeed cruel and unfair to the children and families affected. Tragically, in most known cases, the children have perished from this condition.
We are driven and motivated by a new philosophy. We are taking an active role in supporting scientific efforts to develop a cure for ourselves and others.
Click on the arrow below to learn more about this ultra-rare disease.
Since 2018, Alexander’s Way has been supporting a project to build a personalized genome engineering tool using the CRISPR/Cas9 technology. The goal is to silence or correct the mutation which is responsible for MFM6.
Dr. Jong-Min Lee, at the Massachusetts General Hospital and Harvard Medical School, is leading the group which is currently engaged in doing this work.
Marco's and Alexander's families are advocating and working together to overcome this disease. We will turn hope into reality for children and young adults with this condition.
With your support our organization will sponsor scientists directly, ensuring that resources are used in an effective way.
For the sake of our children and others...Please join us in these efforts !