Children leave hospital after gene therapy trial success
The Children's Hospital of Fudan University has announced a major breakthrough in the treatment of spinal muscular atrophy type 1 (SMA) through a successful clinical trial of a groundbreaking gene therapy. SMA is a rare and severe genetic disorder that causes muscle atrophy due to the degeneration of lower motor neurons.
The hospital reported that the first two children to receive the innovative gene therapy in the trial, including one from Vietnam, have been discharged after undergoing intravenous injection and subsequent follow-up tests to assess safety and effectiveness. This marks a significant milestone as the Vietnamese child is the first expatriate participant in China's gene therapy clinical trial for SMA.
Medical professionals involved in the trial observed positive improvements in both children without encountering serious drug-related complications, highlighting the therapy's efficacy and safety.
The Vietnamese child's inclusion in the trial was made possible by the proactive efforts of Dr. Wang Yi and the clinical trial team, who expedited the screening, preparation, and administration processes to accommodate the child's age, which was close to the trial's age criteria cutoff.
The gene therapy, known as EXG001-307, developed domestically in China, represents a groundbreaking single-dose gene-replacement therapy specifically designed for SMA.
Dr. Wang emphasized the significance of the expat child's participation, stating that it has broadened the accessibility of China's SMA gene treatment trial to children globally. This achievement not only contributes to advancing rare disease research in China but also accelerates new drug trials and facilitates their eventual market availability.
The hospital announced that the clinical trial is actively recruiting patients of all nationalities interested in participating. Those interested can contact Dr. Zhu at 18101843510 for more information.
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