Our team has reached a moment we believe marks an important milestone for children living with spinal muscular atrophy (SMA). In this video, our specialist shares the achievement, and one family shares their journey.
For many years, children with SMA who tested positive for AAV9 antibodies particularly at high titres were considered ineligible for certain gene therapy approaches. We're proud to say this has now changed.
Following careful patient selection, thorough evaluation, meticulous planning, and close monitoring throughout, our team has successfully treated three children with SMA who were AAV9 antibody-positive with high titres. All three children underwent the procedure safely.
This achievement is about far more than three patients. It challenges a long-held assumption and opens the door to new scientific possibilities for children who may previously have been considered ineligible for gene therapy.
"My name is Ruslan, and I am the father of a child with SMA. After speaking with doctors, we came to Fakeeh University Hospital in Dubai. From the moment we arrived, the medical team welcomed us, listened to us, and took excellent care of our child. We are deeply grateful to the doctors, nurses, translators, and the entire team who supported us throughout this journey."
Our sincere thanks go to our incredible multidisciplinary team, our international collaborators, and, above all, the courageous families who placed their trust in us. This is not the end of the journey it's the beginning of a new chapter in gene therapy. We remain committed to collecting robust scientific data, sharing our experience with the global medical community, and continuing to advance what may be possible for children living with neuromuscular diseases.
The future of gene therapy is being shaped today, and we are honoured to be part of that story.
