Updates:
Dear friends!
To date, we managed to fundraise for a half of treatments. We still need about 7000 pounds! We still ask you to support and spread this request.
Muscular dystrophies are a group of hereditary, neurological-muscular conditions that cause a progressive weakening of muscles. There is no effective cure for it. The only available forms of partial treatment are a steroid therapy and a regular physical therapy. However the effects of this therapies are barely noticeable.
The most promising results among experimental treatments are observed in genetic therapy and stem cell therapy. While the former is still only in a research phase, using mesenchymal stem cell to stop the progression of the disease is already possible and is performed through the transplant of these cells.
In Poland any treatment using MSC (mesenchymal stem cell) injection has to be approved by the Bioethical Commision. The transplants are performed by KLARA Medical Center in Częstochowa, which is licensed for treatment in specified types of neurological conditions. Mesenchymal cells are obtained from Wharton’s jelly and are prepared by the Institute of Cellular Engineering of the Polish Stem Cell Bank S.A. with the approval of the General Pharmaceutical Agency. Read more Mesenchymal stem cells are known for their unique regenerative qualities. They can replicate during long periods of time and unlike other cell types can multiply indefinitely. Beside the ability to self-regenerate, they can also differentiate into specialised cells performing concrete functions. The first cell transplant took place in 2014. The patients who underwent the therapy showed a significant improvement, in muscular mass and strength as well as motoric functions. In majority of the cases the treatment slowed down or even stopped the development of the disease. Bioethcial Commision approval and breakdown of the financing of the stem cell transplant (in Polish)