SMA: One drug administration makes it possible for patients and their families to forget about the disease

During a meeting of the Parliamentary Group on SMA, experts presented data on the benefits that gene therapy has brought to patients. Children who received it as part of the drug program, even before the onset or severity of the disease's symptoms, they stressed, are now developing virtually as well as their healthy peers.

More than 4,000 children with spinal muscular atrophy worldwide are being treated with gene therapy. - The longest follow-up published is 7.5 years. The results of ongoing long-term observations in patients with pre-symptomatic and symptomatic SMA indicate that a single intravenous administration of the therapy provides sustained high efficacy and, importantly, with no new safety signals," said Prof. Mazurkiewicz-Beldzinska, and noted the success of implementing a screening program and treatment for SMA. - I absolut...