Latest Treatment Updates
Gene Therapy Fundraising Campaign
We are excited to announce that we have identified a promising gene therapy treatment for Tas called Elevedys. This innovative treatment targets the underlying genetic cause of DMD and has shown encouraging results in patients who have received it.
Elevedys was approved in June 2024 for ambulatory, genetically confirmed patients aged four years and older suffering from DMD. It is essentially an infusion product that prompts the body to produce microdystrophin, which can stop the destruction of muscle cells.
The treatment costs $3.5 million, which is why we are launching this fundraising campaign. We are inspired by the success story of a community in Hungary that successfully raised funds for a little boy from Debrecen to receive this treatment last summer.
Help Fund Tas's Treatment
Therapy Progress
Tas continues with his rigorous therapy schedule: three times a week for kinesiotherapy, once for movement therapy, and once for therapeutic massage. His therapists have noted his determination and resilience during these sessions.
The specialized exercises and stretching routines are crucial for maintaining muscle flexibility and function for children with DMD. Tas has been diligent with his home exercises as well, showing incredible determination despite the challenges.
We've also introduced swimming when possible, which has been particularly beneficial as it allows Tas to move more freely in the water while still building strength. He absolutely loves his pool sessions and looks forward to them whenever possible.
Fundraising Campaign Launch
We are officially launching our fundraising campaign to raise $3.5 million for Tas's gene therapy treatment. This is an ambitious goal, but we believe that with the support of our community and beyond, we can make this life-changing treatment accessible to Tas.
We want to express our heartfelt gratitude to everyone who has donated, shared our campaign, or supported us in any way. Your generosity is making a real difference in Tas's life and giving him hope for the future.
Special thanks to our initial donors who have helped us kickstart this campaign. Every contribution, no matter the size, brings us closer to our goal.
Medication Update
Tas continues to take Deflazacort, a steroid that we import from Germany as it is not yet approved in Romania. This medication, along with his regular therapy sessions, is crucial for slowing the progression of his DMD.
While the steroids come with side effects—Tas is shorter than his peers, can concentrate for less time, and is more irritable and hyperactive—they are currently the only treatment that helps preserve his muscle integrity.
We continue to monitor his response closely and are grateful for the expert care provided by his medical team. We are also researching additional treatments and therapies that might benefit Tas as we work toward our goal of accessing gene therapy.