NEW HYDE PARK, Long Island (PIX11) — Advances in medical research are leading to the development of new drugs that are helping to lift people out of the ravages of disease.
The introduction of a new drug is improving the life of a young Massapequa teenager.
Her medical improvement was celebrated at the Cohen Children’s Medical Center in New Hyde Park, Long Island.
Daniella Sawyers marked her 17th birthday at her home last June. As an infant, she was diagnosed with Rett syndrome, a rare genetic neurological disorder that occurs in one out of every 10,000 female births.
“Patients with Rett Syndrome have a sense of impairment in life, an inability to speak, to walk, to eat,” according to Dr. Sanjeev Kothare, a pediatric neurologist.
“It’s almost like being trapped behind glass. She wants to express much, and she can’t do it,” Sawyers’ mother explained.
But much of that is changing due to a new drug called Daybue, approved by the FDA last March. At the Cohen Children’s Medical Center, doctors and her parents celebrated the progress Daniella has made with the drug in just a period of months.
“She engages more, more in focus, puts words together stronger, and her gait is a little stronger,” her mother has noticed.
Daniella’s eyes glistened, and her smile expressed her happiness when the Consul General of Barbados surprised her with the answer to her long-standing dream to visit the Caribbean island.
“We’re going to fly the family to Barbados,” Mackie Holder revealed.
Though the new drug is helping Daniella, its yearly treatment is costly at an estimated quarter of a million dollars. The family said that, for now, insurance is covering the cost. As long as Daniella remains on this treatment, doctors are optimistic about her future. Kothare said he hopes it will improve her cognition, communication skills, and gait.
Daniella has a long road ahead, but thanks to medical advances, she is slowly emerging from the darkness of a rare disease. Right now, she’s looking forward to realizing a lifelong dream to visit the birthplace of her mother on the sunny beaches of Barbados.