KJ Muldoon, a 9 1/2-month-old boy with CPS1 deficiency, received a groundbreaking custom gene-editing treatment, becoming the first patient of any age to do so. Despite the grim prognosis for his condition, his parents chose to pursue this innovative approach instead of palliative care. This pioneering treatment is expected to have far-reaching implications for over 30 million Americans affected by rare genetic disorders, highlighting the need for more development in gene therapy for these conditions.
The implications of the treatment go far beyond treating KJ, said Dr. Peter Marks, who was the Food and Drug Administration official overseeing gene-therapy regulation until he recently resigned over disagreements with Robert F. Kennedy Jr., the secretary of health and human services.
KJ has made medical history. The baby, now 9 1/2 months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors.
We loved him, and we didn't want him to be suffering, said Muldoon. But she and her husband decided to give KJ a chance.
More than 30 million people in the United States have one of more than 7,000 rare genetic diseases. Most are so rare that no company is willing to spend years developing a gene therapy that so few people would need.
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