Gene Replacement Therapy for MLD

Metachromatic Leukodystrophy (MLD) is a rare and progressive disorder that affects the nervous system and causes severe neurological decline. There is currently no cure for MLD, and existing treatments are limited and not always effective. The emotional and financial burden of caring for a loved one with MLD is immense, and the need for new treatments is urgent.

Dr. Escolar is a respected researcher in the field of gene therapy, and her team has been working diligently to develop a safe and effective treatment for this disorder. The trial has been carefully designed to meet all ethical and safety guidelines, and we are confident in the potential for success. However, funding for rare disease clinical trials is very limited and the trial is on pause due to lack of funding. This is devastating for patients and their families that are struggling with the adverse progression of this devastating disease.

Any donation, no matter the amount, would be greatly appreciated and will help us to move closer to a cure for this disorder.