Batten-Disease-Information-Page

Batten Disease Information Page


What research is being done?

Much of the NINDS research on Batten disease focuses on developing a better understanding of the disease, gene therapy, and the development of drugs to treat the disorder. Scientists are using a modified safe virus to deliver a functioning gene to the brain with hopes the replacement gene will take over or restore the mutated gene's normal function. Using an animal model, NINDS-funded scientists are combining gene therapy with bone marrow transplant as a therapy for infantile Batten disease.  NIH researchers have identified a potential new drug to treat infantile Batten disease that uses a non-toxic molecule to prevent the harmful cellular residue buildup. Among other research, scientists are looking at the development of new molecules to treat Batten and a variety of neurological disorders. Other scientists hope to identify biomarkers--biological signs that may indicate the presence or progression of a disease--to better understand the disease and possibily develop new treatments. The NINDS helps fund the Lysosomal Diseases Network, a combined network of research centers, clinical investigators, patient advocacy groups, and other interested parties that advocate for research on diagnosing, managing, and treating lysosomal and related diseases, including Batten disease.

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What research is being done?

Much of the NINDS research on Batten disease focuses on developing a better understanding of the disease, gene therapy, and the development of drugs to treat the disorder. Scientists are using a modified safe virus to deliver a functioning gene to the brain with hopes the replacement gene will take over or restore the mutated gene's normal function. Using an animal model, NINDS-funded scientists are combining gene therapy with bone marrow transplant as a therapy for infantile Batten disease.  NIH researchers have identified a potential new drug to treat infantile Batten disease that uses a non-toxic molecule to prevent the harmful cellular residue buildup. Among other research, scientists are looking at the development of new molecules to treat Batten and a variety of neurological disorders. Other scientists hope to identify biomarkers--biological signs that may indicate the presence or progression of a disease--to better understand the disease and possibily develop new treatments. The NINDS helps fund the Lysosomal Diseases Network, a combined network of research centers, clinical investigators, patient advocacy groups, and other interested parties that advocate for research on diagnosing, managing, and treating lysosomal and related diseases, including Batten disease.

Much of the NINDS research on Batten disease focuses on developing a better understanding of the disease, gene therapy, and the development of drugs to treat the disorder. Scientists are using a modified safe virus to deliver a functioning gene to the brain with hopes the replacement gene will take over or restore the mutated gene's normal function. Using an animal model, NINDS-funded scientists are combining gene therapy with bone marrow transplant as a therapy for infantile Batten disease.  NIH researchers have identified a potential new drug to treat infantile Batten disease that uses a non-toxic molecule to prevent the harmful cellular residue buildup. Among other research, scientists are looking at the development of new molecules to treat Batten and a variety of neurological disorders. Other scientists hope to identify biomarkers--biological signs that may indicate the presence or progression of a disease--to better understand the disease and possibily develop new treatments. The NINDS helps fund the Lysosomal Diseases Network, a combined network of research centers, clinical investigators, patient advocacy groups, and other interested parties that advocate for research on diagnosing, managing, and treating lysosomal and related diseases, including Batten disease.


Definition
Definition
Treatment
Treatment
Prognosis
Prognosis
Clinical Trials
Clinical Trials
Organizations
Organizations
Publications
Publications
2780 Airport Drive, Suite 342
Columbus, OH 43219
Tel: 800-448-4570
Children's Brain Disease Foundation[A Batten Disease Resource]
Parnassus Heights Medical Building, Suite 900
Suite 900
San Francisco, CA 94117
Tel: 415-665-3003
6475 East Pacific Coast Highway
Suite 466
Long Beach, CA 90803
Tel: 877-621-1122
459 State Road 135 South
Greenwood, IN 46142
Tel: 317-888-7396