As a pediatrician and advocate for children’s health, I recognize that gene therapies for rare conditions like spinal muscular atrophy or Duchenne muscular dystrophy are often held up as evidence of the promise of genetic engineering.
While some of these therapies have shown potential in some rare clinical conditions, they do not justify the uncontrolled expansion of genetic manipulation across medicine, agriculture, and biotechnology.
These technologies remain largely experimental, with significant gaps in our understanding of off-target effects, immune activation, epigenetic disruption, and long-term outcomes, especially in vulnerable populations like children, whose developing systems are far more susceptible to genomic manipulations.
Pointing to isolated medical successes cannot be used to deflect our concerns about the unregulated and unpredictable nature of gene editing platforms now infiltrating everything from vaccines to infant nutrition. The process of scientific inquiry and questioning must not be dismissed, gaslit, or obfuscated, especially when the health and futures of our children are at stake.
It is understood that the conversation around genetic engineering technologies must be nuanced, acknowledging both their potential and their risks, especially when these powerful tools are extended into the clinical setting from its birthplace in agriculture, where long-term consequences still remain unknown despite decades of use.
For those of us who have been concerned with the genetic modification of our food and medicine, our voices have been largely silenced or ignored
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder that primarily affects boys, occurring in approximately 1 in every 3,500 to 5,000 male births worldwide.
Caused by mutations in the dystrophin gene, which is essential for muscle strength and stability, DMD leads to gradual muscle degeneration and weakness, typically beginning in early childhood.
Most children with DMD lose the ability to walk by their early teens and face life-threatening complications such as cardiomyopathy and respiratory failure as the disease progresses.
DMD remains a devastating diagnosis with a significantly shortened life expectancy, often into the late twenties or early thirties. Early diagnosis and multidisciplinary care can improve quality of life and slow disease progression, but a cure has not yet surfaced.
For parents of children with DMD, Sarepta Therapeutics’ gene therapy, Elevidys (delandistrogene moxeparvovec), was
touted as a groundbreaking treatment.
Approved by the US FDA in June 2023 under the accelerated approval pathway, Elevidys was the first gene therapy authorized for DMD, initially targeting ambulatory patients aged 4 to 5 years with a confirmed mutation in the DMD gene.
In June 2024, the FDA expanded Elevidys’ approval to include ambulatory and non-ambulatory individuals aged 4 and older, based on evidence of micro-dystrophin expression which is a protein essential for muscle function. However, this expansion was granted under accelerated approval for non-ambulatory patients, contingent upon further clinical verification of the therapy’s benefit
Of note, the net revenues for the first quarter for Sarepta Therapeutics totaled $611.5 million, a 70% increase over the same quarter of the previous year.
Additionally, a single treatment of Elevidys costs wholesale approximately $3.2 million
for a one-time intravenous infusion, $2.4 million for those with Medicaid, making it one of the most expensive therapies in the world.
Medical Genetic Engineering and
Our Children’s Safety
Dr. Michelle Perro, MD, Pediatrician
She has worked as both Emergency Room Director and Attending Physician from New York’s Metropolitan Hospital/New York Medical college to UCSF Benioff Children’s Hospital, Oakland, CA. Dr. Perro has co-authored the highly acclaimed book, What’s Making our Children Sick. Stay tuned for her upcoming book, Making our Children Well, an instructional guidebook for parents on nutrition and homeopathy.