On July 20, the Gieselmann family walked through the doors of Le Bonheur Children’s Hospital for 5-year-old Elle’s 23rd dose of Brineura™ (cerliponase alfa). The enzyme replacement therapy, approved by the U.S. Food and Drug Administration (FDA) in April, is the family’s only hope for slowing the progression of Elle’s Batten disease – a rare diagnosis that leads to progressive loss of motor and cognitive functions and seizures. Batten disease claimed the life of Elle’s older sister, Milla, last year.

Until the drug’s recent FDA approval for neuronal ceroid lipofuscinosis  (CLN type 2) Batten disease, Elle participated in its clinical trial at Nationwide Children’s Hospital in Columbus, Ohio. The Gieselmanns, who live in Memphis, are relieved Elle can now receive Brineura™– administered twice a month  – close to home. Le Bonheur is one of first hospitals in the country to offer Brineura™.

“Being able to stay home will not only help Elle and her recovery from infusions, but it is a game changer for our family as we continue fighting through each day,” said Elle’s dad, Frazer.

Le Bonheur is committed to helping families who face a rare, devastating diagnosis find hope, says Neuroscience Institute Co-director James Wheless, MD. The hospital is also one of only a handful of children’s hospitals to offer Spinraza™, a groundbreaking new drug for spinal muscular atrophy – a rare and often fatal genetic disease that affects muscular strength and the child’s ability to move and even breathe.

”Offering innovative therapies for children facing rare diseases is important, particularly when they have few other options,” Wheless said.  “At Le Bonheur, we have all the resources in place to deliver cutting-edge treatment options to families.”