Gene therapy broke a barrier this year with the introduction of Zolgensma, an adeno-associated viral vector-based gene therapy for children with spinal muscular atrophy. This type of gene therapy will likely be the first option to treat Aashni's mitochondrial disease as well as many other people with the various types of mitochondiral disease and dysfunction. The only problem is that Novartis is charging over $2 million for this treatment. The United Mitochondrial Disease Foundation uses donated money to help advance research in these treatments and provides support to patients and families with mitochondrial disease.
Aashni is continuing to fight the odds and thrive despite the limitations her disease places on her body. She will be starting kindergarten in the fall and is excited that she will stay with her teacher and all of her friends from T-K. Aashni loves singing songs, reading books, and taking baths with her younger brother Aarav. Please help support the UMDF and all the babies, kids, teenagers and adults that they support. We are hoping for a therapy like Zolgensma to be made for Aashni and other Polg G patients. A monetary donation is greatly appreciated but even sharing with friends and family helps spread the word!!
Running the Bank of America Chicago Marathon is the pinnacle of achievement for elite athletes and everyday runners alike. On race day, runners from all 50 states and more than 100 countries will set out to accomplish a personal dream by reaching the finish line in Grant Park. The UMDF's Team Activate has participated in the marathon for the past 8 years as a charity partner, and we are excited for our 9th year as a partner with this world-class event.