FDA Approves first Treatment for a Form of Batten Disease
[Source: Medical News Today]
The U.S. Food and Drug Administration has approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
“The FDA is committed to approving new and innovative therapies for patients with rare diseases, particularly where there are no approved treatment options,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research. “Approving the first drug for the treatment of this form of Batten disease is an important advance for patients suffering with this condition.”
PediaStaff is Hiring!All Jobs
PediaStaff hires pediatric and school-based professionals nationwide for contract assignments of 2 to 12 months. We also help clinics, hospitals, schools, and home health agencies to find and hire these professionals directly. We work with Speech-Language Pathologists, Occupational and Physical Therapists, School Psychologists, and others in pediatric therapy and education.