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Late neurosurgeon acclaimed for groundbreaking discoveries, impacting thousands of lives

Dr. Andrew Freese was a neurosurgeon and pioneer in gene therapy.

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Myrtelle Announces Successful Completion of Initial Stage of Phase 1/2 Clinical Trial of Proprietary Gene Therapy for Canavan Disease and Expands Treatment to Younger Patients

New gene therapy trial in US offers hope for children with Canavan disease

0 shares Benny Landsman, 4, at Dayton Children’s Hospital April 8, 2021, before surgery to receive the first clinical trial of a new gene therapy for Canavan disease. (Courtesy of the Landsman family/ via Dayton Jewish Observer) Benny Landsman, 4, is prepped for surgery at Dayton Children’s Hospital, April 8, 2021. (Courtesy of the Landsman family/ via Dayton Jewish Observer) Benny Landsman with Paola Leone and Dr. Christopher G. Janson at Dayton Children’s Hospital, April 8, 2021. (Courtesy of the Landsman family/ via Dayton Jewish Observer) Jordana Holovach with her late son, Jacob, who died of Canavan disease at age 19. (Courtesy of Jordana Holovach/ via Dayton Jewish Observer)

Gene Therapy Trial Offers Hope for Children With Canavan

By Marshall Weiss, The Dayton Jewish Observer (reprinted with permission) | May 06, 2021 In an operating room at Dayton Children’s Hospital on April 8, 4-year-old Benny Landsman of Brooklyn received the first new clinical trial of an FDA-approved gene therapy for Canavan disease. Neurosurgeon Dr. Robert Lober placed four catheters in holes drilled into Benny’s skull, down a track to a fluid space in the brain’s ventricles. Lober then manually injected a fluid containing 370 trillion viral genomes of the new therapy. By his side in the operating room were the trial’s clinical leader, Dr. Christopher G. Janson, also with Dayton Children’s and Premier Health’s Clinical Neuroscience Institute, and longtime Canavan researcher Paola Leone, professor of cell biology at Rowan University in Glassboro, New Jersey, and director of Rowan’s Cell and Gene Therapy Center.

Family raises millions for treatment of sons' rare and fatal neurological disorder

While the Landsman family is optimistic that the gene therapy will help their two sons, they also hope it will help families not receive same dire prognosis.

Niños dibujaron un mundo con vacunas que derrotan al covid