Lipid-Storage-Diseases-Information-Page

Lipid-Storage-Diseases-Information-Page

Lipid Storage Diseases Information Page

What research is being done?

The National Institute of Neurological Disorders and Stroke (NINDS) has made significant contributions to research on lipid storage diseases and their treatment. NINDS researchers were responsible for developing effective enzyme replacement therapies for Gaucher and Fabry diseases. NINDS-funded scientists continue to study how lipids accumulate in cells and why they cause harm to the body. Researchers hope to develop new treatments, including gene- and cell-based therapies.

The NINDS, along with other Institutes and Centers of the National Institutes of Health, supports the Lysosomal Disease Network, a network of centers that address some of the major challenges in the diagnosis, management, and therapy of rare diseases, including the lipid storage diseases.

Information from the National Library of Medicine’s MedlinePlus
Metabolic Disorders

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What research is being done?

The National Institute of Neurological Disorders and Stroke (NINDS) has made significant contributions to research on lipid storage diseases and their treatment. NINDS researchers were responsible for developing effective enzyme replacement therapies for Gaucher and Fabry diseases. NINDS-funded scientists continue to study how lipids accumulate in cells and why they cause harm to the body. Researchers hope to develop new treatments, including gene- and cell-based therapies.

The NINDS, along with other Institutes and Centers of the National Institutes of Health, supports the Lysosomal Disease Network, a network of centers that address some of the major challenges in the diagnosis, management, and therapy of rare diseases, including the lipid storage diseases.

Information from the National Library of Medicine’s MedlinePlus
Metabolic Disorders

The National Institute of Neurological Disorders and Stroke (NINDS) has made significant contributions to research on lipid storage diseases and their treatment. NINDS researchers were responsible for developing effective enzyme replacement therapies for Gaucher and Fabry diseases. NINDS-funded scientists continue to study how lipids accumulate in cells and why they cause harm to the body. Researchers hope to develop new treatments, including gene- and cell-based therapies.

The NINDS, along with other Institutes and Centers of the National Institutes of Health, supports the Lysosomal Disease Network, a network of centers that address some of the major challenges in the diagnosis, management, and therapy of rare diseases, including the lipid storage diseases.

Information from the National Library of Medicine’s MedlinePlus
Metabolic Disorders


Definition
Definition
Treatment
Treatment
Prognosis
Prognosis
Clinical Trials
Clinical Trials
Organizations
Organizations
Publications
Publications

Niemann-Pick disease information sheet compiled by the National Institute of Neurological Disorders and Stroke (NINDS).

Metachromatic Leukodystrophy information sheet compiled by the National Institute of Neurological Disorders and Stroke (NINDS).