Nemours Children's Health to Host Gene Therapy Clinical Trial for Patients with Rare Disorder

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Nemours Children’s Health will host a gene therapy clinical trial for a rare genetic condition.

Nemours was chosen by the Foundation for the National Institutes of Health Accelerating Medicines Partnership as one of eight sites to conduct clinical trials. Nemours trials will be for Morquio A Syndrome.

Morquio A affects 1 in 200,000, and it’s caused by an inherited gene mutation.

It can cause serious complications including cervical spinal cord compression, short stature, flat feet, difficulty walking, hearing loss, tracheal obstruction, and heart valvular disease

Dr. Shunji Tomatsu has held National Institutes of Health grant funding for Morquio Syndrome since 2010, and along with his colleagues identified gene therapy that could be the answer for patients.

Dr. Tomatsu is also the head of the Skeletal Dysplasia Research Lab with Nemours.

"We are very happy Morquio A Syndrome was chosen by the NIH committee. This is huge for sure to move forward with a clinical trial, and then in the near future we might get approval for a drug,” said Tomatsu. “This is an astonishing milestone for sure."

The goal is to start enrollment for the clinical trial in 2024.

He’s excited to move forward with gene therapy.

"I'm hopeful for Morquio families, and patients, they have waited for a long time for this opportunity so therefore we are very pleased to move forward," said Tomatsu.

Tomatsu is a Nemours principal investigator that has studied Morquio A for 35 years with National Institutes of Health funding for the last 12 years.

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