Pantothenate kinase-associated neurodegeneration
Other Names for this Disease
- Hallervorden-Spatz disease
- Neuroaxonal dystrophy, late infantile
- Neurodegeneration with brain iron accumulation
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Currently there is no cure for this condition. Treatment consists of medications and surgery to relieve symptoms. For many of the treatments that do improve symptoms, the period of benefit is limited. Baclofen and trihexyphenidyl remain the most effective drugs for the dystonia and spasticity associated with this condition. Botulinum toxin may be helpful for many affected individuals, especially in treating a limited body region. For example, injections in the facial muscles can greatly improve speech and eating abilities. Those with PKAN typically do not benefit from L-dopa. Deep brain stimulation (DBS) is also an option for relieving some symptoms; an international study of the effectiveness of DBS is currently underway. Recently, interest in chelating agents (agents that remove iron from the body) has also been revived, although the benefits have not yet been documented and systemic anemia remains a risk. A trial using deferriprone (a chelator) in PKAN is currently underway in Italy. Click on the link to learn more about this study.
Last updated: 9/23/2011
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- ClinicalTrials.gov lists trials that are studying or have studied Pantothenate kinase-associated neurodegeneration. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.