Currently, no widely accepted treatment can cure, slow down, or halt the symptoms in the great majority of patients with<unk> , but seizures may be controlled or reduced with use of antiepileptic drugs. Additionally, physical, speech, and occupational therapies may help affected patients retain functioning for as long as possible. Several experimental treatments are under investigation. Cystagon In 2001, a drug used to treat cystinosis, a rare genetic disease that can cause kidney failure if not treated, was reported to be useful in treating the infantile form of<unk> . Preliminary results report the drug has completely cleared away storage material from the white blood cells of the first six patients, as well as slowing down the rapid neurodegeneration of infantile<unk> .Currently, two drug trials are underway for infantile<unk> , both using Cystagon. Gene therapy A gene therapy trial using an adenoassociated virus vector called AAV2CUhCLN2 began in June 2004 in an attempt to treat the manifestations of late infantile<unk> . The trial was conducted by Weill Medical College of Cornell University and sponsored by the Nathan's Battle Foundation. In May 2008, the gene therapy given to the recipients reportedly was "safe, and that, on average, it significantly slowed the disease's progression during the 18-month follow-up period" and "suggested that higher doses and a better delivery system may provide greater benefit". A second gene therapy trial for late infantile<unk>  using an adenoassociated virus derived from the rhesus macaque (a species of Old World monkey) called AAVrh.10 began in August 2010, and is once again being conducted by Weill Medical College of Cornell University. Animal models of late infantile<unk>  showed that the AAVrh.10 delivery system "was much more effective, giving better spread of the gene product and improving survival greatly". A third gene therapy trial, using the same AAVrh.10 delivery system, began in 2011 and has been expanded to include late infantile<unk>  patients with moderate tosevere impairment or uncommon genotypes, and uses a novel administration method that reduces general anesthesia time by 50% to minimize potential adverse side effects. Flupirtine Flupirtine A painkiller available in several European countries, flupirtine, has been suggested to possibly slow down the progress of<unk> , particularly in the juvenile and late infantile forms. No trial has been officially supported in this venue, however. Currently, the drug is available to<unk>  families either from Germany, Duke University Medical Center in Durham, North Carolina, or the Hospital for Sick Children in Toronto. Stem cells On October 20, 2005, the Food and Drug Administration approved a phase-I clinical trial of neural stem cells to treat infantile and late infantile Batten disease. Subsequent approval from an independent review board also approved the stem cell therapy in early March 2006. This treatment will be the first transplant of fetal stem cells performed on humans. The therapy is being developed by Stem Cells Inc and is estimated to have six patients. The treatment was to be carried out in Oregon. Juvenile<unk>  has recently been listed on the Federal Clinical Trials website to test the effectiveness of bone-marrow or stem-cell transplants for this condition. A bone-marrow transplant has been attempted in the late infantile form of<unk>  with disappointing results; while the transplant may have slowed the onset of the disease, the child eventually developed the disease and died in 1998. Trials testing the effectiveness of bone-marrow transplants for infantile<unk>  in Finland have also been disappointing, with only a slight slowing of disease reported. Immunosuppressants In late 2007, Dr. David Pearce et al. reported that Cellcept, an immunosuppressant medication commonly used in bone-marrow transplants, may be useful in slowing down the progress of juvenile<unk> . Enzyme replacement therapy On April 27, 2017, the U.S. FDA approved cerliponase alfa as the first specific treatment for<unk> .
Neuronal ceroid lipofuscinosis
Tay–Sachs disease
Pantothenate kinase-associated neurodegeneration
Infantile neuronal ceroid lipofuscinosis
Glycogen storage disease
Granulovacuolar degeneration
Cerebral creatine deficiency
Familial encephalopathy with neuroserpin inclusion bodies