News

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

Events

COVID-19 Mitigation Strategies in Pediatric Rare Disease Clinical Trials Virtual Workshop – May 7th, 2021 (4/21/2021) - A virtual workshop "COVID-19 Mitigation Strategies in Pediatric Rare Disease Clinical Trials" is being offered by the Critical Path Institute. On May 7, 2021, 12:00-1:30 PM Eastern Time (US), the Patient-Reported Outcome (PRO) Consortium’s Rare Disease Subcommittee will hold a complimentary virtual workshop on COVID-19 Mitigation Strategies in Pediatric Rare Disease Clinical Trials. The workshop facilitators… Continue Reading

Research

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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