A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem
Source: Orphanet Journal of Rare Diseases
Unveiling the untreated: development of a database algorithm to identify potential Fabry disease patients in Germany
Source: Orphanet Journal of Rare Diseases
Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains
Source: Orphanet Journal of Rare Diseases
Genome-wide determinants of mortality and motor progression in Parkinson’s disease
Source: npj Parkinson's Disease
Neither alpha-synuclein fibril strain nor host murine genotype influences seeding efficacy
Source: npj Parkinson's Disease
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