Pilot Projects of the LYSOSOMAL DISEASE NETWORK (LDN) (5/14/2021) - The Lysosomal Disease Network (LDN), a consortium organized under the NIH Rare Diseases Clinical Research Network program, aims to improve the treatment of lysosomal diseases by learning more about each of these conditions by funding a variety of projects including 1-year pilot studies. LDN intends to fund 1-2 pilot projects, on an overlapping annual basis,… Continue Reading


Letter from Sue Kahn, Executive Director, NTSAD: Starting a new chapter after stepping down from leadership (6/7/2021) - National Tay-Sachs & Allied Diseases AssociationJune 2021   Dear NTSAD Community,    It's with mixed emotions that I share with you that after nearly 14 years, I am stepping down from my role as NTSAD’s executive director at the end of October. I am looking forward to starting a new chapter in my life, knowing that NTSAD remains strong. I will… Continue Reading


Consensus recommendations for the classification and long-term follow up of infants who screen positive for Krabbe Disease – new PubMed article! (5/12/2021) - Consensus recommendations for the classification and long-term follow up of infants who screen positive for Krabbe Disease.Thompson-Stone R, Ream MA, Gelb M, Matern D, Orsini JJ, Levy PA, Rubin JP, Wenger DA, Burton BK, Escolar ML, Kurtzberg J.Mol Genet Metab. 2021 Apr 3:S1096-7192(21)00083-4. doi: 10.1016/j.ymgme.2021.03.016. Online ahead of print.PMID: 33832819  Excerpt - "Objective: to provide updated evidence and consensus-based recommendations for… Continue Reading

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