Loss of mfsd8 alters the secretome during Dictyostelium aggregation
Source: European Journal of Cell Biology
Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications
Source: Orphanet Journal of Rare Diseases
Nonhuman primate genetic models for the study of rare diseases
A novel porcine model of CLN3 Batten disease recapitulates clinical phenotypes
Source: DMM Disease Models and Mechanisms
Anti-N-Methyl-D-Aspartate Receptor Encephalitis Presenting Atypically as a Mimic of Rapidly Progressive Neurodegenerative Disorder in a Toddler: A Case Report
Source: Pediatric Neurology
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