NIH-funded LDN Investigators Meeting

The Council of Research Experts (CORE) – also known as the NIH-funded Investigators, or Lysosomal Disease Network Investigators – met on Friday, March 4, 2016. Presentation slides from the meeting can be found here.

Fellowship Applications Are Now Open

The Lysosomal Disease Network invites applications for the LDN Fellowship. Applications must be submitted by the deadline of 5:00 P.M. CST Wednesday June 1, 2016. Click here for more information.

RFA for New Pilot Projects

The Lysosomal Disease Network (LDN) announces a new Pilot Project program designed to increase intra-consortium research endeavors.

Applications will be accepted from all Investigators meeting the criteria. Applications are due June 1, 2016. Click here for more information.

Lysosomal Disease Network

Lysosomal diseases are a collection of more than 70 clinical syndromes with incidence rates ranging from1 in 20,000 (Gaucher disease) to 1 in 300,000 (Wolman disease) live births; taken together these conditions are responsible for a significant amount of disability and disease burden.

Testing New Therapies

The rarity of each lysosomal disease means that no single medical research center has an opportunity to see sufficient numbers of patients with any one disease to effectively describe the full spectrum of each disease or adequately test any new therapies.

Combined and Integrated Efforts

The combined and integrated efforts of the LDN focus on creating a network of centers with expertise in one or more of these diseases in order to solve major challenges in diagnosis, disease management, and therapy. Solutions to these problems will have a direct impact on patients suffering from lysosomal disease and important implications for medical practice.

Lysosomal diseases:

aspartylglucosaminuria; Batten disease; cholesteryl ester storage disease; cystinosis; Danon disease; Fabry disease; fucosidosis; galactosialidosis types I, II & III; Gaucher disease types I, II & III; GM1 gangliosidosis (infantile, juvenile, adult-onset); Krabbe disease; alpha-mannosidosis types I & II; beta-mannosidosis; metachromatic leukodystrophy; mucolipidosis types II, III, & IV; mucopolysaccharidosis types I, II, III, IV, and VI (Hurler, Hurler–Scheie, and Scheie; Hunter, Sanfilippo, Morquio, and Maroteaux–Lamy syndromes, respectively); neuronal ceroid lipofuscinosis (infantile, late infantile, juvenile, adult); Niemann–Pick disease; Pompe disease; Sandhoff disease (infantile, juvenile); Schindler disease types I & II; sialidosis types I & II; Tay-Sachs disease (infantile, juvenile and late-onset); Wolman disease.