Batten Disease Treatment has significantly improved over the years with the U.S. FDA approving enzyme replacement therapy

Batten Disease, also known as Neuromuscular Optic Neuritis, is a progressive and life-threatening neurological illness that typically affects children. In children, Batten Disease can result in the complete loss of the ability to walk. It can also result in permanent nerve damage, leading to severe, permanent impairment of motor function. Due to these serious consequences, children diagnosed with Batten Disease must receive early and aggressive treatment by an experienced, professional neurosurgery specialist.

There are four primary forms of Batten Disease treatments. Two of them involve chemical medications and one involves iontophoresis. Chemical treatments of Batten diseases are usually used to relieve symptoms, such as muscle spasms, pain, and itching that occur with the disease. Commonly used medications for these treatments include doxycycline, cisplatin, chloroquine, hydrocitrate, lactic acid, phenytoin, piroxicam, potassium nitrate, potassium phosphate, thalidomide, tetracycline, and intravenous immunoglobulin. These batten disease treatments must be used cautiously, as prolonged use can lead to severe adverse side effects, especially if other medications are taken concurrently. Recently, in September 2020, Abeona Therapeutics Inc. partnered with Taysha Gene Therapies to boost ABO-202 as potential gene therapy for infantile Batten disease.